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Pipeline News and Updates
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Update for August 27, 2020

8/27/2020

 
Regulatory Update
 
The FDA approved ofatumumab (Kesimpta, Novartis) on 8/21/2020, for the treatment of adults with relapsing multiple sclerosis. Ofatumumab is administered monthly via an auto injector pen. Novartis has set WAC at $83,000 per year. Ofatumumab has the same mechanism of action as ocrelizumab (Ocrevus, Roche), which is administered by intravenous infusion every six months and has an annual WAC of $65,000. Ofatumumab (Arzerra, Novartis) was initially approved as an intravenous infusion in 2009 for the treatment of chronic lymphocytic leukemia.
 
The FDA rejected valoctocogene roxaparvovec and said that at least two years of safety and efficacy data from the ongoing Phase III trial would be needed to support the sustainability of the reduced/eliminated Annualized Bleeding Rate. Three-year data from a Phase I/II trial suggested that factor VIII levels seemed to fall off after 12 to 18 months. This could potentially require additional doses to sustain efficacy, so the FDA requested additional data in a larger population. The Phase III trial is expected to be concluded in November 2021, suggesting a 2020 resubmission of the valoctocogene roxaparvovec BLA.
 
The FDA rejected filgotinib due to concerns over toxicity with the 200 mg dose. The FDA requested data from the ongoing MANTA and MANTA-RAy trials, which are not expected to be completed until 2021.  That would potentially delay resubmission of filgotinib until mid-2021. The trials are evaluating whether filgotinib 200 mg affects sperm production. All approved JAK inhibitors (baricitinib, tofacitinib and upadacitinib) have a Black Box Warning regarding an increased risk for serious infections, lymphomas and thrombosis. It is expected that filgotinib would also have this warning.
 
The FDA accepted the NDA for casimersen for the treatment of Duchenne muscular dystrophy with genetic mutations that are amenable to skipping exon 45 of the Duchenne gene and set a PDUFA date for 2/25/2021.
 
BioMarin submitted an NDA for vosoritide as a treatment for children with achondroplasia.
 
Announced Research Updates
 
Immunicum announced that in the 88 patient, Phase II, open-label, MERECA trial, treatment with ilixadencel plus sunitinib resulted in a 43% overall survival rate compared to 33% with sunitinib alone in patients with renal cell carcinoma.
 
Novartis announced that in the 568 patient, Phase III, COMBI-i Trial, spartalizumab added to dabrafenib and trametinib did not improve progression-free survival compared to dabrafenib and trametinib alone in patients with unresectable or metastatic BRAF V600 mutation-positive cutaneous melanoma.
 
Onconova announced that in the 360 patient, Phase III INSPIRE trial, treatment with rigosertib plus best supportive care did not improve overall survival (OS) compared to physician’s choice chemotherapy (6.4 vs 6.3 months) in patients with high-risk myelodysplastic syndrome.
 
Published Research Updates
 
In a 24-week, 254 patient, Phase III trial, teneligliptan lowered HbA1C by 0.95% compared to a 0.14% decrease with placebo in Chinese patients with type 2 diabetes mellitus inadequately controlled with diet and exercise.
 
In a 6-month, 58 patient, Phase II trial, adding cediranib to docetaxel plus prednisone did not improve progression-free survival in patients with metastatic castrate-resistant prostate cancer.
 
In the 24-week, 65 patient, Phase III, ELIPSE HoFH trial, treatment with evinacumab resulted in a 47.1% reduction in LDL compared to a 1.9% increase with placebo in patients with homozygous familial hypercholesterolemia (HoFH) that remained uncontrolled despite aggressive therapy.
 
24-month interim data from 8 patients enrolled in a 36-month, Phase II, open-label trial, suggested that treatment with Oxalobacter formigenes decreased or normalized plasma oxalate levels in patients with PH type 1 and early end-stage renal disease.
 
In a 36 patient, Phase I, open-label trial, treatment with andecaliximab added to gemcitabine and nab-paclitaxel resulted in progression-free survival of 7.8 months and an objective response rate of 44.4% in patients with pancreatic adenocarcinoma.
 
In the 6-month, 538 patient, Phase III ENLIGHTEN-2 trial, patients treated with olanzapine plus samidorphan had a 4.21% weight gain compared to 6.59% gain with olanzapine monotherapy in patients with stable schizophrenia.

2020 YTD New Drug Approvals – Heading toward a record year?

8/25/2020

 
The FDA approved twenty four new drugs during the first six months of 2020. Another twelve new drugs received approval through August 18th. The FDA reported approval of 48 novel investigational drugs in 2019, lower than the 59 approved in 2018, but still the third highest total in the past 25 years. There are another twenty-seven drugs due for approval (drugs with PDUFA Dates) before the end of the year making this year’s total potential approvals an all-time record of sixty-three new drugs approved.
 
Read on to review the list of 2020 FDA approvals covering a wide range of indications and some budget busters. We’ve included data about availability and pricing if it has been published. Pharmaceutical Pipeline Tracker subscribers are able to generate similar reports at any frequency they choose with much more detail, including safety and efficacy, from the knowledgebase if desired – a great tool for preparing for the P&T Committee meeting.
 
  1. The FDA approved avapritinib (Ayvakit, Blueprint Medicines Corp) on 1/9/20 for the treatment of unresectable or metastatic gastrointestinal stromal tumors (GIST) that harbor a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation. WAC for avapritinib is $32,000 per month.
  2. The FDA approved teprotumumab (Tepezza, Horizon Therapeutics) on 1/21/2020, for the treatment of thyroid eye disease, a rare condition where the muscles and fatty tissues behind the eye become inflamed, causing the eyes to be pushed forward and bulge outwards (proptosis). Horizon has set WAC for teprotumumab at $14,900 per vial. A full 6 month course of treatment will require 23 vials for a total treatment WAC of $343,000 and an estimated price after discounts of $200,000. Teprotumumab was approved 6 weeks early, the original PDUFA date was 3/8/2020. The FDA gave the drug an Orphan Drug Priority Designation. Tuesday’s approval represents the first drug approved for the treatment of thyroid eye disease.
  3. The FDA approved tazemetostat (Tazverik, Epizyme) on 1/23/20, for the treatment of metastatic or locally advanced epithelioid sarcoma not eligible for complete resection. Tazemetostat is given orally twice a day. Epizyme set WAC at $65.68 for a one 200 mg tablet.
  4. The FDA approved the oral peanut-protein immunotherapy, AR101 (Palforzia, Aimmune Therapeutics) on 1/31/2020 for the reduction of allergic reaction incidence and severity in patients aged 4-17 years old with a peanut allergy.
    1. Palforzia was approved with a REMS program requiring special training for prescribers and the initial dosing and each dose escalation to be done in a healthcare setting. It is also required that parents or caregivers be counseled on the need to have injectable epinephrine available.
    2. Aimmune set WAC for Palforzia at $890 per month or $10,680 per year.
    3. ICER released a review of AR101 and DBV Technologies (DBVT)’s Viaskin Peanut in July 2019. ICER expressed concern regarding the lack of long-term data for safety and effectiveness, desensitization did not eliminate the need to be cautious with peanut exposure, and increase in allergic reaction requiring epinephrine was seen during clinical trials.
    4. The panel also did not find evidence to support AR101 and Viaskin Peanut to have a benefit over avoidance of peanuts or non-commercialized oral immunotherapy.
    5. In a pharmacoeconomic analysis, ICER estimated a benchmark fair-market price of $4,800-7,200 per year for Palforzia and $3,000-$4,500 per year for Viaskin Peanut.
  5. The FDA approved lactitol (Pizensy, Braintree Laboratories) on 2/13/2020 for the treatment of chronic idiopathic constipation in adults.
  6. The FDA approved bempedoic acid (Nexletol, Esperion) on 2/21/20 as an adjunct to diet and maximally tolerated statin therapy for the treatment of adults with heterozygous familial hypercholesterolemia (HeFH) or established atherosclerotic cardiovascular disease (ASCVD). Esperion set WAC for bempedoic acid at $11 per day. Esperion is offering a prescription reduction program that will reduce the copay to $10 for up to a 3-month supply.
  7. The FDA approved eptinezumab (Vyepti, Lundbeck) on 2/21/20 for migraine prevention in adults. Eptinezumab is administered as a 30-minute IV infusion every 3 months. Lundbeck priced WAC for eptinezumab at $1,495 for one 100 mg vial.
  8. The FDA approved meloxicam injection (Anjeso, Baudax Bio) for the treatment of moderate to severe pain in adults, as monotherapy or concomitantly with other non-NSAIDs. Meloxicam injection is given as a once-daily intravenous injection and WAC is $94 for one 30 mg vial.
  9. The FDA approved rimegepant (Nurtec ODT, Biohaven) on 2/27/2020 for the acute treatment of migraine with or without aura in adults. Biohaven set WAC for rimegepant at $106.25 for one 75mg tablet. Rimegepant will compete with the triptans, ubrogepant, and lasmiditan. The gepants (ubrogepant and rimegepant) and lasmiditan do not cause vasoconstriction and may be an alternative when patients do not tolerate triptans. Merck abandoned work on two early gepants due to concerns over elevation in transaminase levels with telcagepant, but ubrogepant and rimegepant have not demonstrated liver toxicity.
  10. The FDA approved amisulpride (Barhemsys, Acacia Pharma) on 2/26/2020 for the prevention and treatment of PONV in adult patients. WAC for amisulpride is $21.25 for one 2.5 mg vial.
  11. The FDA approved isatuximab (Sarclisa, Sanofi) on 3/2/2020 to be given in combination with pomalidomide (Revlimid, Celgene) and dexamethasone for the treatment of multiple myeloma in patients that received two or more prior therapies including lenalidomide and a proteasome inhibitor. Sanofi set WAC for a 5 ml vial of isatuximab at $650 and $3,250 for a 25 ml vial.
  12. The FDA approved osilodrostat (Isturisa, Novartis) on 3/6/2020 for the treatment of adults with Cushing's disease who either are candidates for pituitary gland surgery or in whom the disease persists after surgery. WAC was set at $110 for one 1 mg tablet, $400 for one 5 mg tablet and $475 for one 10 mg tablet.
  13. The FDA approved ozanimod (Zeposia, Bristol Myers Squibb) on 3/25/2020 for the treatment of adults with relapsing forms of multiple sclerosis (RMS), including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease. BMS announced it would delay the launch for an unspecified time due to the COVID-19 pandemic. Pricing will be announced shortly before launch. WAC for ozanimod is $236 for one 0.92 mg capsule.
  14. The FDA approved pemigatinib (Pemazyre, Incyte) on 4/17/2020 for the treatment of resistant advanced cholangiocarcinoma. WAC for pemigatinib is $1,214.29 for one 4.5 mg, 9 mg or 13.5 mg tablet.
  15. The FDA approved tucatinib (Tukysa, Seattle Genetics) on 4/17/2020 the treatment of adults with advanced HER2-positive breast cancer who have received at least one prior line of therapy. WAC has been set at $18,500 per month; with a treatment duration of 6-months, a course of treatment will be $111,000 per patient.
  16. The FDA approved sacituzumab govitecan (Trodelvy, Immunomedics) on 4/22/2020 to treat resistant metastatic triple-negative breast cancer. Trodelvy has a boxed warning about the potential risks for severe diarrhea and neutropenia. WAC for sacituzumab govitecan is $4,012.50 for one 180 mg vial.
  17. The FDA approved opicapone (Ongentys, Neurocrine Bioscience) on 4/27/2020, as add-on therapy to levodopa/carbidopa in patients with Parkinson's disease experiencing "off" episodes.
  18. The FDA approved selpercatinib (Retevmo, Lilly) on 5/8/2020, for the treatment of non-small cell lung cancer, advanced medullary thyroid cancer and advanced RET fusion-positive thyroid cancer. The FDA requires identification of a RET gene alteration must be determined before initiating treatment. Selpercatinib is taken twice a day until the disease progresses or there is unacceptable toxicity. Lilly set WAC for a 30-day supply of selpercatinib at $20,600. Selpercatinib is the first drug approved to treat cancers with mutation or fusion in the RET gene, but the drug will eventually have competition from pralsetinib, a second RET fusion mutation therapy from Blueprint Medicines. Blueprint filed an NDA for pralsetinib in 1Q2020, so a competitor for selpercatinib may be available in late 2020 or early 2021.
  19. The FDA approved capmatinib (Tabrecta, Novartis) on 5/6/2020, for the treatment of metastatic non-small cell lung cancer (NSCLC) whose tumors have a mutation that leads to mesenchymal-epithelial transition (MET) exon 14 skipping as detected by an FDA-approved test. Novartis has set WAC for a 28-day supply of capmatinib at $17,950. Capmatinib is the first drug approved to treat cancers with MET exon 14 skipping alterations. The drug will eventually have competition from tepotinib, a MET inhibitor being developed by EMD Serono.
  20. The FDA approved ripretinib (Qinlock, Deciphera Pharmaceuticals), on 5/18/2020, for the treatment of advanced gastrointestinal stromal tumors (GISTs). WAC for ripretinib was set at $355.55 for one 50 mg tablet.
  21. The FDA approved artesunate (Amivas LLC), on 5/26/2020, for the treatment of severe malaria in adult and pediatric patients. Until Amivas transitions artesunate's distribution through wholesalers, healthcare providers treating patients with severe malaria should call CDC to obtain IV artesunate. The CDC Malaria Hotline (770-488-7788) is available Monday–Friday, 9 am–5 pm EST. Outside these hours, providers should call 770-488-7100 and ask to speak with a CDC Malaria Branch clinician.
  22. The FDA approved inebilizumab-cdon (Uplizna, Viela Bio) on 6/11/2020 for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. WAC was set for inebilizumab at $4,367 for one 10 mg vial.
  23. The FDA approved lurbinectedin (Zepzelca, Jazz Pharma/PharmaMar) on 6/16/2020 for the treatment of metastatic small cell lung cancer that has progressed during or after treatment with platinum-based chemotherapy. WAC was set for lurbinectedin at $6,633 for one 4 mg vial.
  24. The FDA approved triheptanoin (Dojolvi, Ultragenyx) on 6/30/2020 as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD). Ultragenyx set WAC for triheptanoin at $4,875 per 500 ml vial, which indicates an average annual cost of $138,000.
  25. The FDA has approved remimazolam (Byfavo, Acacia) on 7/2/2020 for the induction and maintenance of procedural sedation in adults undergoing surgical procedures lasting 30 minutes or less.
  26. The FDA approved fostemsavir (Rukobia, ViiV Healthcare), on 7/3/2020, in combination with other antiretrovirals, for the of treatment heavily treated adults with multidrug-resistant HIV-1 infection failing their current antiretroviral regimen due to resistance, intolerance, or safety considerations. WAC for fostemsavir is $127.50 for one 600 mg ER tablet.
  27. The FDA approved the oral combination of decitabine and cedazuridine (Inqovi, Astex Pharmaceuticals), on 7/7/2020, for treatment of myelodysplastic syndrome or chronic myelomonocytic leukemia.
  28. The FDA approved brexucabtagene autoleucel (Tecartus, Gilead Sciences), on 7/24/2020, for the treatment of relapsed or refractory mantle cell lymphoma. Brexucabtagene autoleucel was approved with a boxed warning and REMS program due to the risks of CRS and neurologic toxicities. Brexucabtagene autoleucel is a one-time treatment and Gilead has priced the drug at $373,000, the same price as its other CAR-T cell therapy axicabtagene ciloleucel (Yescarta).
  29. The FDA approved abametapir (Xeglyze, Dr. Reddy's Laboratories), on 7/24/2020, for the treatment of head lice.
  30. The FDA approved tafasitamab-cxix (Monjuvi, Morphosys & Incyte), on 7/31/2020. Tafasitamab-cxix was approved in combination with lenalidomide for second-line treatment of relapsed/refractory diffuse large B-cell lymphoma.
  31. he FDA approved belantamab mafodotin (Blenrep, GlacoSmithKline), on 8/6/2020 for the treatment of heavily treated relapsed or refractory multiple myeloma. Belantamab mafodotin was approved with a REMS program that requires monitoring and physician and patient education about the ocular risks associated with treatment. In the pooled safety population, ocular adverse reactions occurred in 77% of the 218 patients. The ocular adverse events seen most often include keratopathy (76%), changes in visual acuity (55%), blurred vision (27%), and dry eye (19%). GSK has set WAC at $8,277 per vial for an estimated monthly cost of $23,900.
  32. The FDA approved risdiplam (Evrysdi, Genentech), on 8/7/2020, for treatment of spinal muscular atrophy (SMA) in adults and children 2 months of age and older. Risdiplam will be available in late August through Accredo Health Group, an Express Scripts specialty pharmacy for direct delivery to patients’ homes. Risdiplam will compete with nusinersen (Spinraza, Biogen), which was approved in 2016. Risdiplam has weight and age based dosing until a child weighs 44 pounds. For patients that weigh 44 pounds or more, the dose is standardized to 5 mg per day. At 5 mg per day, Genentech has priced risdiplam at $340,000 per year. For an infant weighing 15 pounds, the annual cost would be less than $100,000. Both risdiplam and nusinersen increase production of survival motor neuron (SMN) protein. However, nusinersen is administered by intrathecal injection under anesthesia, while risdiplam is an oral liquid. WAC for nusinersen is $750,000 during the first year and $375,000 for each subsequent year. Onasemnogene abeparvovec (Zolgensma, Novartis) is a single-dose gene therapy for SMA, but the drug has a price of over $2 million and a Black Box Warning regarding liver toxicity.
  33. The FDA approved nifurtimox (Lampit, Bayer), on 8/7/2021, for the treatment of Chagas disease (American Trypanosomiasis) caused by Trypanosoma cruzi (T. cruzi) in pediatric patients. The two drugs approved to treat Chagas disease are nifurtimox and benznidazole. Benznidazole was approved in 2017. Bayer plans to offer a program to reduce co-pays to $0.
  34. The FDA approved oliceridine (Olinvyk, Trevena), on 8/7/2021 for short-term intravenous use in hospitals or other controlled clinical settings for the management of moderate to severe acute pain in adults. Oliceridine is an opioid and was approved with the opioid boxed warning about addiction, abuse and misuse; life-threatening respiratory depression; neonatal opioid withdrawal syndrome; and risks from concomitant use with benzodiazepines or other central nervous system depressants. Oliceridine is administered intravenously and has a maximum recommended daily dose limit of 27 milligrams.
  35. The FDA approved viltolarsen (Viltepso, NS Pharma), on 8/12/2020, for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation in the DMD gene amenable to exon 53 skipping. Viltolarsen will compete with golodirsen (Vyondys 53, Sarepta), which was approved in 2019 for the treatment of DMD amenable to exon 53 skipping. Efficacy has not been established with either drug. Both will require a study to demonstrate they can improve the time to stand in DMD patients. If the trials fail to prove efficacy, the FDA may start proceedings to withdraw approval. WAC for viltolarsen was set at $1,410 for one 5 ml vial, 50 mg/ml.
  36. The FDA approved satralizumab (Enspryng, Genentech), on 8/17/2020, for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adults who are anti-aquaporin-4 or AQP4 antibody-positive.
 
The unexpected costs of budget-busting new drugs are difficult to manage. A best practice for formulary management is to proactively monitor the pharmaceutical pipeline and review new drugs BEFORE they become available. A decision can be made on whether to add the drug or enact a therapeutic interchange. Unbiased information can be communicated to the medical staff regarding place in therapy, availability and/or restrictions. 
 
Why should someone use the Pharmaceutical Pipeline Tracker to augment current processes to monitor new drug developments? Rather than reviewing multiple data feeds, separating the wheat from the chaff, and preparing all the necessary summary data, Prescribe Right can do that work for you. 
 
Our 1,100+ Informational sources generate daily intelligence about the pharmaceutical pipeline from clinical journals, FDA announcements, medical conference summaries, presentations, industry newsletters, and pharmaceutical company announcements. 
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Search an individual drug as well as lists of drugs by indication, drug class, therapy classification, manufacturer and PDUFA Date. EXPLORE 878 INVESTIGATIONAL DRUGS IN THE PHARMACEUTICAL PIPELINE TRACKER INCLUDING 29 IN LINE FOR APPROVAL BEFORE MID 2021. SUBSCRIBE, THEN USE ANY OF THE 10 SEARCH BUTTONS TO ACCESS DETAILED MONOGRAPHS AND CITATION LINKS, ALL FOR ONLY $339 FOR THE REMAINDER OF 2020 Click here for our economical subscription rates. Our best rate is less than $3 per day!

Update for August 18, 2020

8/18/2020

 
​Regulatory Update
 
The FDA approved viltolarsen (Viltepso, NS Pharma), on 8/12/2020, for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation in the DMD gene amenable to exon 53 skipping. Viltolarsen will compete with golodirsen (Vyondys 53, Sarepta), which was approved in 2019 for the treatment of DMD amenable to exon 53 skipping. Efficacy has not been established with either drug. Both will require a study to demonstrate they can improve the time to stand in DMD patients. If the trials fail to prove efficacy, the FDA may start proceedings to withdraw approval.
 
The FDA approved satralizumab (Enspryng, Genentech), on 8/17/2020, for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adults who are anti-aquaporin-4 or AQP4 antibody-positive. 
 
An FDA review of Mesoblast’s remestemcel-L data questioned the use of a single arm study design and felt the overall response rate used to determine efficacy was too low. However, the FDA’s Oncologic Drugs Advisory Committee voted 8-2 to recommend approval of remestemcel-L for the treatment of pediatric patients with steroid-refractory acute graft-versus-host disease (GVHD). 
 
The FDA accepted the NDA for G1 Therapeutics’ trilaciclib to preserve bone marrow and immune system function in patients being treated with chemotherapy for small cell lung cancer and set a PDUFA date of 2/15/2021.
 
The FDA accepted the BLA for Protalix BioTherapeutics’ pegunigalsidase alfa for the treatment of adult patients with Fabry disease and set a PDUFA date of 1/27/2021.
 
The FDA accepted the NDA for TG Therapeutics’ umbralisib for the treatment of follicular lymphoma and marginal zone lymphoma patients who have received at least one prior anti-CD20 based regimen. A PDUFA date of 2/15/21 has been set.
 
Announced Research Updates
 
Fulcrum reports interim data from 29 patients enrolled in the 48-week, 80 patient, Phase IIb ReDUX4 trial, where treatment with losmapimod did not reduce DUX4-driven gene expression in affected skeletal muscle biopsies at 16-weeks, compared to placebo in patients with facioscapulohumeral muscular dystrophy. Despite the lack of effect in an interim analysis, Fulcrum plans to continue the ReDUX4 trial, because in patients with the highest pre-treatment DUX4-driven gene expression, losmapimod reduced DUX4-driven gene expression more than placebo.
 
Verona announced that in a 4-week, 416 patient, Phase IIb, dose ranging trial, ensifentrine plus tiotropium increased FEV1 compared to tiotropium alone (ensifentrine dose-dependent increase of 78 mL to 124 mL) in patients with chronic obstructive pulmonary disease. 
 
Novo halted the Phase II explorer5 trial and the Phase III explorer7 and 8 trials, in March 2020, due to thrombotic events in three patients. All three trails were evaluating concizumab as prophylactic treatment in both hemophilia A and B regardless of inhibitor status. Novo restarted all three trials in August 2020 with new safety measures in place.
 
Published Research Updates
 
In the 24-week, 2,691 patient, Phase III, SAKURA 3 trial (a long -term follow-up with patients that participated in the SAKURA 1 & 2 trials), treatment with daxibotulinumtoxinA resulted in a reduction in the Investigator Global Assessment-Frown Wrinkle Severity (IGA-FWS) score, which peaked at two to four weeks where 96% of patients had an IGA-FWS score of none to mild and at 24-weeks, 32% of patients had a similar score. The most common ADR were headache (5.9%), nasopharyngitis (4.4%), injection site pain (3.9%), and injection site erythema (3.3%).

Update for August 11, 2020

8/11/2020

 
Regulatory Update
 
The FDA approved belantamab mafodotin (Blenrep, GlacoSmithKline), on 8/6/2020 for the treatment of heavily treated relapsed or refractory multiple myeloma. Belantamab mafodotin was approved with a REMS program that requires monitoring and physician and patient education about the ocular risks associated with treatment. In the pooled safety population, ocular adverse reactions occurred in 77% of the 218 patients. The ocular adverse events seen most often include keratopathy (76%), changes in visual acuity (55%), blurred vision (27%), and dry eye (19%). GSK has set WAC at $8,277 per vial for an estimated monthly cost of $23,900.
 
The FDA approved risdiplam (Evrysdi, Genentech), on 8/7/2020, for treatment of spinal muscular atrophy (SMA) in adults and children 2 months of age and older. Risdiplam will be available in late August through Accredo Health Group, an Express Scripts specialty pharmacy for direct delivery to patients’ homes. Risdiplam will compete with nusinersen (Spinraza, Biogen), which was approved in 2016. Risdiplam has weight and age based dosing until a child weighs 44 pounds. For patients that weigh 44 pounds or more, the dose is standardized to 5 mg per day. At 5 mg per day, Genentech has priced risdiplam at $340,000 per year. For an infant weighing 15 pounds, the annual cost would be less than $100,000. Both risdiplam and nusinersen increase production of survival motor neuron (SMN) protein. However, nusinersen is administered by intrathecal injection under anesthesia, while risdiplam is an oral liquid. WAC for nusinersen is $750,000 during the first year and $375,000 for each subsequent year.  Onasemnogene abeparvovec (Zolgensma, Novartis) is a single-dose gene therapy for SMA, but the drug has a price of over $2 million and a Black Box Warning regarding liver toxicity.
 
The FDA approved nifurtimox (Lampit, Bayer), on 8/7/2021, for the treatment of Chagas disease (American Trypanosomiasis) caused by Trypanosoma cruzi (T. cruzi) in pediatric patients. The two drugs approved to treat Chagas disease are nifurtimox and benznidazole. Benznidazole was approved in 2017. Bayer plans to offer a program to reduce co-pays to $0.
 
The FDA approved oliceridine (Olinvyk, Trevena), on 8/7/2021 for short-term intravenous use in hospitals or other controlled clinical settings for the management of moderate to severe acute pain in adults. Oliceridine is an opioid and was approved with the opioid boxed warning about addiction, abuse and misuse; life-threatening respiratory depression; neonatal opioid withdrawal syndrome; and risks from concomitant use with benzodiazepines or other central nervous system depressants. Oliceridine is administered intravenously and has a maximum recommended daily dose limit of 27 milligrams.
 
The FDA rejected the Viaskin Peanut desensitizing patch due to concerns over patch adhesion and how it affects efficacy. The FDA requested additional manufacturing data, a redesign of the patch adhesion and a new trial to demonstrate efficacy with the new patch adhesion.
 
The EMA conditionally approved MYR Pharmaceuticals’ bulevirtide for the treatment of chronic hepatitis D with compensated liver disease.
 
Announced Research Updates

Elios Therapeutics announced that in a 3-year, 144 patient, Phase IIb trial, treatment with TLPLDC resulted in disease free survival (DFS) in 51.8% of patients and overall survival (OS) in 92.9% of patients compared to a DFS of 27.1% and OS of 70.3% with placebo in patients with Stage III or Stage IV melanoma at high risk of recurrence following complete surgical resection.
Genentech announced that in the 10-week, 358 patient, Phase III, HIBISCUS I trial, treatment with etrolizumab induced remission in more patients than placebo in patients with moderately to severely active ulcerative colitis who have not been previously treated with anti-TNF agents. However, in the 10-week, 358 patient, Phase III, HIBISCUS II trial, treatment with etrolizumab was no different than placebo in inducing remission in patients with moderately to severely active ulcerative colitis who have not been previously treated with anti-TNF agents. Genentech announced that in the 14-week, 609 patient, Phase III, HICKORY trial, more patients treated with etrolizumab had a successful induction compared to placebo, but etrolizumab was no better than placebo in sustaining remission in patients with moderately to severely active ulcerative colitis who have been previously treated with anti-TNF agents. Genentech announced that in the 62-week, 359 patient, Phase III, LAUREL trial, etrolizumab was no better than placebo in sustaining remission in patients with moderately to severely active ulcerative colitis who have not been previously treated with anti-TNF agents.
 
Published Research Updates
 
41 heavily pretreated patients with relapsed or refractory Hodgkin’s lymphoma were enrolled in two Phase I/II trials and received CD 30 CAR-T cell therapy. One-year progression-free survival was 36% and overall survival was 94%. The overall response rate in 32 patients with active disease who received fludarabine-based lymphodepletion was 72%.
 
In a 12-week, 352 patient, Phase IIb, dose ranging trial, treatment with fezolinetant reduced the Menopause-Specific Quality of Life questionnaire VMS scores by 2.9 to 4.4 compared to a 2.3 point decrease with placebo, reduced the Hot Flash-Related Daily Interference Scale by 3.3 to 4.3 points compared to 2.9 point decrease with placebo and reduced Greene Climacteric Scale-VMS domain scores by 2.1 to 3.3 points compared to a 2.1 point decrease with placebo in patients with moderate to severe vasomotor symptoms.

Update for August 4, 2020

8/4/2020

 
Regulatory Update
 
The FDA approved tafasitamab-cxix (Monjuvi, Morphosys & Incyte), on 7/31/2020. Tafasitamab-cxix was approved in combination with lenalidomide for second-line treatment of relapsed/refractory diffuse large B-cell lymphoma.
 
The FDA accepted the BLA for Ridgeback Biotherapeutics’ ansuvimab for the treatment of Ebola virus infections.
 
The FDA has granted Marinus Pharmaceuticals’ ganaxolone a Rare Pediatric Disease Designation for the treatment of CDKL5 deficiency disorder, a rare refractory form of pediatric epilepsy.
 
BMS resubmitted a BLA for idecabtagene vicleucel for the treatment of relapsed and refractory multiple myeloma.
 
CHMP (European new drug review committee) recommended approval of Geron/Janssen’s imetelstat for the treatment of myelodysplastic syndromes.
 
Announced Research Updates
 
Orion announced that in the 12-week, 496 patient, Phase III REFALS trial, treatment with levosimendan did not improve the slow vital capacity in the supine position compared to placebo in patients with ALS. Levosimendan also did not improve patient functionality and survival at 48 weeks.
 
Allergan announced that in the 12-week, 910 patient, Phase III, ADVANCE trial, monthly migraine days were reduced by 3.69 days with atogepant 10 mg, 3.86 days with 30 mg, and 4.2 days with 60 mg compared to a 2.48 days decrease with placebo in patients with four to 14 migraine days per month.
 
In a 6-week, 477 patient, Phase III trial, viloxazine decreased the ADHD-RS-5 total score by 16.6 points with 100 mg and 17.7 points with 200 mg compared to a 10.6 point decrease with placebo in children 6 to 11 years of age with attention deficit hyperactivity disorder. 
 
Published Research Updates
 
In a 492 patient, Phase III trial, adding pegvorhyaluronidase alfa to nab-paclitaxel plus gemcitabine resulted in overall survival of 11.2 months compared to 11.5 months with nab-paclitaxel plus gemcitabine alone in patients with hyaluronan-high metastatic pancreatic ductal adenocarcinoma. 
 
In a 195 patient Phase III trial, tivantinib did not improve progression-free survival compared to placebo in Japanese patients with highly expressed c-Met hepatocellular carcinoma with prior treatment with sorafenib. 
 
In a 12-week, 387 patient, Phase III, JADE MONO-1 trial, 24% of patients treated with abrocitinib 100 mg and 44% treated with 200 mg achieved an IGA score of clear or almost clear skin, had a 2 point or > improvement in their IGA score compared to 8% with placebo in patients with moderate to severe atopic dermatitis. A 75% or greater improvement in the Eczema Area and Severity Index (EASI-75) score was achieved by 40% of patients in the 100 mg group and by 63% in the 200 mg group compared to 12% with placebo.
 
In a 150 day, 1,453 patient, Phase II trial, 2.6% of patients treated with nirsevimab developed medically attended RSV-associated lower respiratory tract infection compared to 9.5% with placebo in healthy infants who had been born preterm (29 weeks 0 days to 34 weeks 6 days of gestation).
 
In a 6-week, 51 Japanese patient, Phase II trial, hemoglobin increased by 0.43 g/dL with vadadustat 150 mg, 1.13 g/dL with 300 mg, and 1.62 g/dL with 600 mg compared to a 0.47 g/dL decrease with placebo in patients with nondialysis-dependent CKD (NDD-CKD) induced anemia. In a 6-week, 60 Japanese patient, Phase II trial, hemoglobin decreased by 0.28 g/dL with vadadustat 150 mg, but increased by 0.08 g/dL with 300 mg, and 0.41 g/dL with 600 mg compared to a 1.48 g/dL decrease with placebo in patients with dialysis-dependent CKD (DD-CKD) induced anemia. By week 16, 91% of NDD-CKD patients and 71% of DD-CKD patients had achieved hemoglobin levels between 10.0 and 12.0 g/dL in both trials.
 
GSK announced topline results from a 52-week, 271 patient, Phase III trial, where oral daprodustat was non-inferior to darbepoetin in raising mean hemoglobin levels (10.9 g/dl vs 10.8 g/dl) during weeks 40 to 52 in dialysis-dependent Japanese patients with anemia associated with chronic kidney disease. 
 
In the 40-week, 76 patient, Phase II, STAIRWAY trial, best-corrected visual acuity was improved by 9.3 letters with faricimab every 12 weeks and by 12.5 letters with every 16 weeks dosing compared to an 11.4 letter improvement with ranibizumab every 4 weeks in patients with neovascular age-related macular degeneration.

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