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

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.

 
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