US FDA approval and panel tracker: June 2023

June was gene therapy month, with FDA approvals for both Sarepta’s Elevidys and Biomarin’s Roctavian. Both have challenges ahead, however. Elevidys was given only accelerated approval in Duchenne muscular dystrophy patients aged 4-5 years old; expanding the label to other ages depends upon the confirmatory Embark trial, data from which are due later in the year. For Roctavian, a haemophilia A gene therapy, durability remains a concern, and many expect a sluggish US launch, mirroring the slow roll out seen in Europe. Several setbacks also occurred in June, with three delays and seven complete response letters. Intercept’s tortuous Nash journey finally ended with another knockback for Ocaliva, while the rejection of Regeneron’s high dose Eylea will allow Roche’s Vabysmo to gain more ground on Eylea’s original formulation. Lastly, all the panels had positive outcomes last month, including one for Eisai and Biogen’s Leqembi, setting the Alzheimer’s therapy up for full approval by 6 July.

Notable first-time US approval decisions in June 2023
Project Company Indication(s) 2028e WW SBI ($m) Outcome
Elevidys (SRP-9001, delandistrogene moxeparvovec) Sarepta/Roche Ambulatory patients with Duchenne muscular dystrophy with a confirmed mutation in the DMD gene


Approved in patients aged 4-5 years old (accelerated)
Roctavian (valoctocogene roxaparvovec, gene therapy) Biomarin Adults with severe haemophilia A  1,430 Approved (previous CRL)
Bimzelx UCB Plaque psoriasis 937 Delayed to Q3
Glofitamab (Columvi) Roche Relapsed/refractory large B-cell lymphoma after ≥2 lines lines of systemic therapy 763 Approved (accelerated, ~ 2 weeks early)
Litfulo (ritlecitinib) Pfizer 12 yrs+ with alopecia 502 Approved
Momelotinib GSK Myelofibrosis with anaemia 465 Delayed to 16 September
Rystiggo (rozanolixizumab) UCB Generalised myasthenia gravis 323 Approved
Olorofim F2G (private)/Shionogi Invasive fungal infections in patients who have limited or no treatment options 21 CRL (additional data and analyses)
ADX-2191 (methotrexate injection) Aldeyra Primary vitreoretinal lymphoma 9 CRL (lack of substantial evidence of effectiveness)
Vyvgart Hytrulo (subcutaneous efgartigimod) Argenx Generalised myasthenia gravis - (IV forecasts 2,598**) Approved
Epinephrine nasal spray (Neffy) ARS Pharmaceuticals Emergency treatment of allergic reactions (type I) including anaphylaxis in adults and children ≥ 30 kg - Delayed to 19 September
Vevye (CyclASol/ cyclosporine ophthalmic solution) Novaliq (subsidiary of Geuder Group) Dry eye disease - Approved
Ocaliva (obeticholic acid) Intercept Pre-cirrhotic liver fibrosis due to Nash - Second CRL (long-term outcomes needed, Intercept discontinuing all-Nash related investment)
Travivo (Exxua) Fabre-Kramer Pharmaceuticals/Bristol Major depressive disorder - Not yet disclosed
Dehydrated alcohol injection (DS-100) Eton Methanol poisoning - CRL (Chemistry Manufacturing and Controls)
IPX203 Amneal Parkinson's disease - CRL (additional PK data)
Lantidra (donislecel, allogeneic pancreatic islet cellular therapy) Celltrans (private) Adults with type 1 diabetes who are unable to approach target glycated haemoglobin because of current repeated episodes of severe hypoglycemia - Approved
Ngenla Pfizer/Opko Paediatric growth hormone deficiency - Approved (previous CRL)
*Forecast prior to adcoms, **MG indication assigned to IV version. SBI: sales by indication. Sources: Evaluate Pharma & company releases.


Advisory committee meetings in June 2023
Project Company Indication 2028e SBI ($m) Outcome
Leqembi Eisai/Biogen Alzheimer's disease (confirmatory study) 4,409 6-0 in favour (Pdufa 6 July)
Beyfortus (nirsevimab) Astrazeneca/ Sanofi/Sobi Prevent RSV lower respiratory tract disease in neonates and infants 1,727 21-0 in favour (newborns or infants born/entering 1st RSV season)
19-2 in favour (up to 24 months of age who remain vulnerable through their 2nd RSV season)
(Pdufa Q3)
Sohonos (palovarotene capsules) Ipsen Prevention of heterotopic ossification in adults and children (females aged ≥8 years and males ≥10 years) with fibrodysplasia ossificans progressiva - 10-4 in favour of effectiveness
11-3 in favour benefits outweigh the risks
(Pdufa 16 August)
- - Covid-19 (vaccine composition) - 21-0 in favour of updating the current vaccine composition to a monovalent XBB-lineage
RSV: respiratory syncytial virus. Sources: FDA ad com calendar, Evaluate Pharma & company releases.
Supplementary and other notable approval decisions in June 2023
Product Company Indication (clinical trial) Outcome
Prevymis Merck Prophylaxis of CMV disease in adult kidney transplant recipients at high risk (NCT03443869) Approved
Linzess Ironwood Patients 6-17 years old with functional constipation (NCT04026113) Approved
Bylvay (odevixibat) Ipsen (Albireo) Alagille syndrome (Assert) Approved
Camzyos (mavacamten) Bristol Myers Squibb Reduce the need for septal reduction therapy in adults with symptomatic obstructive hypertrophic cardiomyopathy (Valor-HCM) Approved
Talzenna + Xtandi Pfizer Adult patients with homologous recombination repair (HRR) gene-mutated metastatic castration-resistant prostate cancer (Talapro-2) Approved
Jardiance, Synjardy Boehringer Additions to diet and exercise to improve blood sugar control in children 10 years and older with type 2 diabetes (Dinamo) Approved
Blincyto Amgen Minimal residual disease-positive B-cell precursor ALL (ECOG-E1910, AALL1331) Conversion to full approval
Eylea 8mg Regeneron/Bayer Wet age-related macular degeneration and diabetic macular oedema (Pulsar, Photon) CRL (ongoing review of inspection findings)
AVT02 (biosimilar) Alvotech/Teva Humira interchangeability CRL (deficiencies following facilities reinspection, previously received two CRLs for biosimilarity BLA)
Gavreto Roche/Blueprint Advanced or metastatic rearranged during transfection (RET)-mutant medullary thyroid cancer who require systemic therapy Withdrawn (not feasible to conduct confirmatory study, AcceleRET-MTC)
Janssen Covid-19 vaccine (Ad26.COV2-S) J&J Prevention of Covid-19 EUA withdrawn
Source: Evaluate Pharma & company releases.

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