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 ($m) Outcome
Amvuttra (vutrisiran) Alnylam Polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults 2,722 Approved
AXS-05 Axsome Major depressive disorder 787 No decision yet, FDA did provide proposed labelling
Priorix GSK MMR vaccine 371 Approved
Tebipenem HBr Spero Complicated urinary tract infections including pyelonephritis 181 CRL (additional clinical study required)
(sodium phenylbutyrate)
Acer Urea cycle disorder - CRL (facility inspection)
Spesolimab Boehringer Ingelheim (private) Generalised pustular psoriasis - No decision yet
AMX0035 Amylyx ALS - Extended to Sep 29 (additional analyses of data)
Annik (penpulimab) Akeso/Sino 3L nasopharyngeal carcinoma - No decision yet (est H1)
Ryzneuta Evive Biotech (private) Chemotherapy-induced neutropenia - No decision yet
SH-111 Shorla Oncology (private) Undisclosed project for T-cell leukaemia - No decision yet
Source: company releases & Evaluate Pharma.
Advisory committee meetings in June
Project Company Indication 2028e SBI ($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/
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
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/
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.

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