US FDA approval tracker: October 2022
Several drugs got a grilling from the FDA in October, none more so than Covis’s Makena, which is used to reduce preterm birth risk. For a second time, an FDA panel recommended that Makena be removed from the market; the project received accelerated approval in 2011 but its confirmatory study failed in 2019. Now the FDA needs to follow through on its advice, with a final decision expected in the coming months. GSK’s Duvroq, a pill to manage anaemia in chronic kidney disease, had a mixed panel, being recommended for patients on dialysis but not for non-dialysis patients. On the approval front Johnson & Johnson gained a green light for Tecvayli in relapsed/refractory multiple myeloma, although the BCMA bispecific has a Rems program similar to Car-T therapies. Gilead, on the other hand, received a knockback for its hepatitis D project Hepcludex due to concerns over manufacturing and delivery. Gilead gained the project through its $1.4bn buyout of Myr two years ago. Meanwhile, Amicus will have to wait even longer for a decision on its Pompe disease candidate, which was delayed again by the FDA's inability to inspect a plant in China.
| Notable first-time US approval decisions in October | ||||
|---|---|---|---|---|
| Project | Company | Indication(s) | 2028e sales by indication ($m) | Outcome |
| Tecvayli (teclistamab) | Johnson & Johnson | R/r multiple myeloma | 1,704 | Approved (accelerated) |
| Hepcludex (bulevirtide) | Gilead/ Hepatera | Chronic hepatitis delta virus infection in adults with compensated liver disease | 639 | CRL (concerns regarding manufacture & delivery) |
| Cipaglucosidase for AT-GAA | Amicus | Pompe's disease | 266* | Deferred again (alongside second component miglustat; lack of manufacturing site inspection) |
| SPN-830 apomorphine infusion device | Supernus | Continuous treatment of motor fluctuations (off episodes) in Parkinson’s disease | 198 | CRL (infusion device issues, inspections) |
| Tislelizumab | Beigene/ Novartis |
2nd-line oesophageal squamous cell carcinoma | 83 | Pending |
| Furoscix | Scpharmaceuticals | Congestion in patients with worsening heart failure | - | Approved |
| Imjudo (tremelimumab) + Imfinzi (Stride regimen) | Astrazeneca | Unresectable hepatocellular carcinoma | - | Approved |
| *For AT-GAA product. Source: Evaluate Pharma & company releases. | ||||
| Advisory committee meetings in October | ||||
|---|---|---|---|---|
| Project | Company | Indication | 2028e SBI ($m) | Outcome |
| Duvroq (daprodustat) | GSK | Anaemia due to CKD in adults on/not on dialysis | 355 | 13-3 in favour for patients on dialysis, 5-11 against in non-dialysis patients |
| Sohonos (palovarotene) | Ipsen | Prevention of heterotopic ossification in adults & children with fibrodysplasia ossificans progressiva | 152 | Postponed (FDA request for new information on clinical trial data) |
| Omblastys (omburtamab) |
Y-Mabs | Neuroblastoma with CNS/leptomeningeal metastases | 69 | 16-0 against, not provided sufficient evidence to conclude that omburtamab improves OS |
| Makena | Amag/ Covis | Reducing risk of preterm birth in women who have a history of singleton spontaneous preterm birth | - | 14-1 that Makena was not effective, and that it should be removed from the market |
| Source: Evaluate Pharma, company releases & FDA adcom calendar. | ||||
| Supplementary and other notable approval decisions in October | |||
|---|---|---|---|
| Product | Company | Indication (clinical trial) | Outcome |
| Oxlumo | Alnylam | Reduction of plasma oxalate in advanced primary hyperoxaluria type 1 (Illuminate-C) | Approved |
| Rinvoq | Abbvie | Non-radiographic axial SpA (Select-Axis 2 Study 2) | Approved |
| Boostrix | GSK | Immunisation during third trimester to prevent pertussis (whooping cough) in infants under 2mths old | Approved |
| Menveo | GSK | Single-vial presentation to prevent disease caused by meningococcal bacteria serogroups A, C, Y & W in individuals aged 10-55 | Approved |
| mRNA-1273.222 (bivalent) | Moderna | Covid vaccine booster dose, down to six years of age | EUA |
| Omicron BA.4/BA.5-adapted bivalent Covid-19 vaccine | Pfizer/Biontech | Covid vaccine booster dose, down to five years of age | EUA |
| Novavax Covid-19 vaccine, adjuvanted (NVX-CoV2373) | Novavax | Covid vaccine booster dose, in adults aged 18 and over (Prevent-19, Cov-Boost) | EUA |
| Source: Evaluate Pharma & company releases. | |||
