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 |
3,357 |
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. | |||
