US FDA approval and panel tracker: March 2023
Last month was a busy one for FDA advisory committee meetings. Following Pfizer’s earlier positive panel with its respiratory syncytial virus vaccine Abrysvo, this time it was GSK’s turn with Arexvy, with its own vote looking more favourable. Several issues such as safety, durability and reimbursement have yet to play out, but both GSK and Pfizer’s vaccines look likely to gain approval in May. The highly competitive space saw Johnson and Johnson shelve its own phase 3 vaccine project in late March. In another adcom, Roche’s Polivy came away with a positive decision in first-line diffuse large B-cell lymphoma. The outcome was a surprise because briefing documents had focused on the lack of a survival benefit, and modest PFS. Polivy's Pdufa was set for 2 April. Lastly, Biogen/Ionis’s tofersen could be heading for accelerated approval in Sod1-mutated ALS after a panel voted unanimously in favour of using data from a controversial biomarker to predict a clinical benefit. Tofersen’s Pdufa is 25 April.
| Notable first-time US approval decisions in March | ||||
|---|---|---|---|---|
| Project | Company | Indication(s) | 2028e SBI ($m) | Outcome |
| Valrox (Roctavian) | Biomarin | Haemophilia A gene therapy | 1,420 | Delayed to June 30 (previously submitted three-year data analysis) |
| ABBV-951 (foscarbidopa/foslevodopa for continuous subcutaneous delivery) | Abbvie | Treatment of motor fluctuations in adults with advanced Parkinson's disease. | 979 | CRL (additional information about the device) |
| Zavzpret (zavegepant) | Pfizer (Biohaven) | Acute treatment of migraine | 562 | Approved |
| Rezzayo (rezafungin) | Cidara/Melinta/ Mundipharma | Candidemia and invasive candidiasis in adults who have limited or no alternative treatment options | 348 | Approved |
| Daybue (trofinetide) | Acadia | Rett | 273 | Approved |
| Joenja (leniolisib) | Pharming/Novartis | Activated phosphoinositide 3-kinase delta syndrome | 189 | Approved |
| Zynyz (retifanlimab) | Incyte | Adults with metastatic or recurrent locally advanced Merkel cell carcinoma | - | Approved (previous CRL in squamous cell carcinoma of the anal canal) |
| SBI: sales by indication. Source: Evaluate Pharma & company releases. | ||||
| Advisory committee meetings in March | ||||
|---|---|---|---|---|
| Project | Company | Indication | 2028e SBI ($m) | Outcome |
| Paxlovid | Pfizer | Mild-to-moderate coronavirus disease in adults who are at high risk for progression to severe Covid-19, including hospitalisation or death |
4,867 | 16 to 1 in favour |
| Arexvy (RSVPreF3 OA) | GSK | Prevention of lower respiratory tract disease caused by RSV-A and RSV-B subtypes in adults 60 years of age and older (recombinant, adjuvanted vaccine) | 1,742 | In favour: efficacy (12-0), safety (10-2) |
| Polivy | Roche | In combo with a rituximab product, cyclophosphamide, doxorubicin, and prednisone for the treatment of adult patients with previously untreated diffuse DLBCL | 1,497* | 11 to 2 in favour |
| Tofersen | Biogen/Ionis | ALS associated with a mutation in the SOD1 gene | 10 | 9 to 0 in favour of using biomarker data to predict clinical benefit in accelerated decision |
| Naloxone hydrochloride nasal spray, 3 mg/0.1 mL (Rivive) | Harm Reduction Therapeutics | Nonprescription use as an opioid reversal agent in the emergency treatment of opioid overdose | - | Cancelled, no longer needed |
| *Already approved in 3L use. Source: Evaluate Pharma, company releases & FDA adcom calendar. | ||||
| Supplementary and other notable approval decisions in March | |||
|---|---|---|---|
| Product | Company | Indication (clinical trial) | Outcome |
| Narcan nasal spray (4 mg/0.1 mL) | Emergent Biosolutions | OTC emergency treatment for known or suspected opioid overdose. | Approved |
| Naloxone hydrochloride nasal spray 4mg | Amphastar | Emergency treatment of known or suspected opioid overdose | Approved |
| Evkeeza | Regeneron | Adjunct to other lipid-lowering therapies to treat children aged 5 to 11 years with homozygous familial hypercholesterolemia (NCT04233918) | Approved |
| Hyrimoz (high concentration formulation of 100 mg/mL) (Humira biosimilar) | Novartis | Includes the indications of the reference medicine Humira | Approved |
| Verzenio + endocrine therapy | Eli Lilly | Adjuvant HR+ Her2- node-positive, early breast cancer at a high risk of recurrence (four-year data from monarchE) | Approved |
| Tafinlar + Mekinist | Novartis | Paediatric patients with BRAF V600E low-grade glioma (Tadpole) | Approved |
| Nelarabine injection (SH-111) | Shorla Oncology (private) | T-cell acute lymphoblastic leukaemia and T-cell lymphoblastic lymphoma | Approved (ANDA) |
| Keytruda | Merck & Co | Adult and paediatric patients with unresectable or metastatic MSI-H or dMMR solid tumours | Conversion to full approval (Keynote-158, -164, -051) |
| Pfizer-BioNTech Covid-19 Vaccine, Bivalent | Pfizer/Biontech | Single booster dose of the vaccine in children 6 months to 4 years old (at least 2 months after completion of primary vaccination) | EUA amended |
| Empaveli injector | Apellis | PNH (on-body drug delivery system that would enable patients to self-administer pegcetacoplan through subcutaneous infusion) | Extended (timing TBC) |
| Lynparza + Zytiga + prednisone/prednisolone | Astrazeneca | Metastatic castration-resistant prostate cancer (Propel) | Delayed, adcom set for 28 April |
| Sabizabulin | Veru | Hospitalised adult patients with moderate to severe Covid-19 who are at high risk for acute respiratory distress syndrome | Declined EUA (negative adcom in November 2022) |
| Makena | Covis (private) | Reducing risk of preterm birth in women who have a history of singleton spontaneous preterm birth | To be withdrawn, follows second negative adcom last Oct |
| QD (once daily) ruxolitinib (Jakafi) extended release formulation | Incyte | Myelofibrosis, polycythemia vera and GVHD | CRL |
| ANDA; abbreviated NDA. Source: Evaluate Pharma & company releases. | |||
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