US FDA approval tracker: June 2022
It was FDA panels galore last month, with good news for most. Bluebird gained recommendations for its gene therapies eli-cel and beti-cel; full approvals will be financially crucial as these would give the group priority review vouchers to sell. FDA adcoms also recommended that Covid vaccines from Moderna and Pfizer/Biontech move into younger populations, with expanded EUAs quickly following. And Novavax finally found favour for its vaccine, although a final EUA decision could be delayed by manufacturing changes. Separately, a panel decided that an Omicron component should be included in future Covid boosters, although there were concerns about the lack of data on specific subvariants. However, it was bad news for Acadia as a panel went against its label expansion push for Nuplazid, a second setback for the antipsychotic. On approvals, Alnylam's more convenient follow-on RNAi project Amvuttra got a thumbs up in amyloidosis polyneuropathy as investors await Onpattro data in cardiomyopathy, a larger indication. Also Merck & Co's 15-valent pneumococcal vaccine Vaxneuvance is now greenlit for use in infants, a large chunk of the market. Pfizer, the leader in this space, will soon report phase 3 infant data on Prevnar 20, which covers an additional five strains.
| Notable first-time US approval decisions in June | ||||
|---|---|---|---|---|
| Project | Company | Indication(s) | 2028e sales by indication ($m) | Outcome |
| Amvuttra (vutrisiran) | Alnylam | Polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults | 2,722 | Approved |
| AXS-05 | Axsome | Major depressive disorder | 787 | No decision yet, FDA did provide proposed labelling |
| Priorix | GSK | MMR vaccine | 371 | Approved |
| Tebipenem HBr | Spero | Complicated urinary tract infections including pyelonephritis | 181 | CRL (additional clinical study required) |
| ACER-001 (sodium phenylbutyrate) |
Acer | Urea cycle disorder | - | CRL (facility inspection) |
| Spesolimab | Boehringer Ingelheim (private) | Generalised pustular psoriasis | - | No decision yet |
| AMX0035 | Amylyx | ALS | - | Extended to Sep 29 (additional analyses of data) |
| Annik (penpulimab) | Akeso/Sino | 3L nasopharyngeal carcinoma | - | No decision yet (est H1) |
| Ryzneuta | Evive Biotech (private) | Chemotherapy-induced neutropenia | - | No decision yet |
| SH-111 | Shorla Oncology (private) | Undisclosed project for T-cell leukaemia | - | No decision yet |
| Source: company releases & Evaluate Pharma. | ||||
| Advisory committee meetings in June | ||||
|---|---|---|---|---|
| Project | Company | Indication | 2028e SBI ($m) | Outcome |
| Spikevax | Moderna | Updates to EUAs for Covid-19 vaccines to include younger populations | 3,214 | Recommended (21-0) (6 mths-5 yrs) Recommended (22-0) (6-17 yrs) |
| Comirnaty | Pfizer/ Biontech |
11,605 | Recommended (21-0) (6 mths-4 yrs) | |
| Nuvaxovid | Novavax | Prevent Covid-19 in individuals 18 years of age and older | 5,766 | Recommended (21-0, with 1 abstention) |
| N/A | N/A | Discuss whether the Sars-CoV-2 strain composition of Covid-19 vaccines should be modified, and if so which strain should be selected for autumn 2022 | - | Recommended inclusion of Omicron component in Covid booster vaccines (19-2) |
| Nuplazid | Acadia | Hallucinations and delusions associated with Alzheimer's disease psychosis | 2,181 | Voted against (9-3) |
| Elivaldogene autotemcel (Skysona, eli-cel) | Bluebird bio | Early cerebral adrenoleukodystrophy (aged under 18) | 21 | Recommended (15-0) |
| Betibeglogene autotemcel (Zynteglo, beti-cel) | Bluebird bio | Beta-thalassaemia (who require regular red blood cell transfusions) | 120 | Recommended (13-0) |
| Source: FDA ad com calendar & Evaluate Pharma. | ||||
| Supplementary and other notable approval decisions in June | |||
|---|---|---|---|
| Product | Company | Indication (clinical trial) | Outcome |
| Beovu | Novartis | Diabetic macular oedema (Kestrel, Kite) | Approved |
| Dupixent | Sanofi/ Regeneron |
Moderate-to-severe atopic dermatitis (6 months to 5 years) (Liberty AD Preschool) | Approved |
| Imcivree | Rhythm | Bardet-Biedl syndrome or Alström syndrome (NCT03746522) | Approved in BBS, CRL in Alström |
| Breyanzi | Bristol | 2L r/r large B-cell lymphoma intended for stem cell transplant (Transform) | Approved |
| Riabni (Rituxan biosmilar) | Amgen | Adults with moderate-to-severe rheumatoid arthritis | Approved |
| Olumiant | Lilly | Alopecia areata (Brave-AA1, Brave-AA2) | Approved |
| Skyrizi | Abbvie | Moderately-to-severe Crohn's disease in adults (Advance, Motivate, Fortify) | Approved |
| Vaxneuvance | Merck & Co | 15 valent pneumococcal vaccine (Pneu-Ped) | Approved (slightly ahead of delayed July date) |
| Tafinlar + Mekinist | Novartis | Tumour-agnostic indication for BRAF V600E solid tumours (Roar, NCI-MATCH Subprotocol H study, Study X2101) | Approved (accelerated) |
| Zulresso | Sage | Include patients 15+ years old with postpartum depression | Approved |
| Reblozyl | Bristol/ Merck & Co |
Anaemia in adults with non-transfusion-dependent beta-thalassaemia (ph2 Beyond) | Withdrawn (company could not appropriately address FDA questions about risk-benefit) |
|
Maxigesic IV |
Hyloris/AFT Pharmaceuticals | Post-op pain | CRL (revise a risk assessment) |
| Source: company releases & Evaluate Pharma. | |||
| FDA Covid-19 EUAs | ||
|---|---|---|
| Product | Company | Outcome |
| Spikevax | Moderna | Include use of the vaccine in individuals 6 months to 17 years of age |
| Comirnaty | Pfizer/Biontech | Include use of the vaccine in individuals 6 months to 4 years of age |
Supplemental table has been updated to include Beovu's approval in DME.
