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.
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.
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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%).
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.
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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