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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