US FDA approval tracker: November 2022
Apellis investors suffered a disappointment last month as the Pdufa for intravitreal pegcetacoplan was pushed out to February. The delay was caused by Apellis submitting longer term data from two geographic atrophy phase 3 studies, only one of which hit the primary 12 month measure. News was better for Immunogen, which gained an accelerated approval for mirvetuximab soravtansine, now called Elahere, in FRα-positive platinum-resistant ovarian cancer. A confirmatory study is already underway with data expected early next year. But the FDA’s crackdown on previous accelerated decisions continued in November, with Roche’s Tecentriq and GSK’s Blenrep and Zejula all being withdrawn in certain oncology indications after confirmatory studies failed. For Ardelyx’s Xphozah there were glimmers of hope after an earlier CRL and two appeals. An FDA adcom, convened in response to the second appeal, voted favourably for the project, which aims to control serum phosphorus in adults with chronic kidney disease on dialysis. The Office of New Drugs now has until mid-December to provide a response to Ardelyx's appeal.
| Notable first-time US approval decisions in November | ||||
|---|---|---|---|---|
| Project | Company | Indication(s) | 2028e SBI ($m) | Outcome |
| Intravitreal pegcetacoplan | Apellis | Geographic atrophy secondary to age-related macular degeneration | 2,567 | Delayed until February (Apellis plans to submit 24-month efficacy data) |
| Tzield (teplizumab) | Provention Bio | Delay the onset of Stage 3 type 1 diabetes in patients aged 8 and older with Stage 2 | 952 | Approved (previous CRL) |
| Elahere (mirvetuximab soravtansine) | Immunogen | Folate receptor alpha-high platinum-resistant ovarian cancer who have been previously treated with 1 to 3 prior systemic treatments | 759 | Approved (accelerated) (confirmatory Mirasol data due early 2023) |
| AT-GAA (cipaglucosidase + miglustat) | Amicus | Pompe disease | 266 | Previous delays, FDA type A meeting now expected before YE |
| Poziotinib | Spectrum | Previously treated Her2 exon 20 insertion mutated NSCLC | 137 | CRL (another study needed) |
| Omblastys (omburtamab) | Y-mAbs Therapeutics | Treatment of neuroblastoma with CNS/leptomeningeal metastases | 69 | CRL (another study needed) |
| Hemgenix (etranacogene dezaparvovec) | Uniqure/CSL | Adults with haemophilia B | - | Approved |
| Rebyota (RBX2660) | Ferring (private) | Reduce the recurrence of Clostridioides difficile infection in adults following antibiotic treatment for recurrent C diff infection | - | Approved |
| SBI: sales by indication. Source: Evaluate Pharma, company releases. | ||||
| Advisory committee meetings in November | ||||
|---|---|---|---|---|
| Project | Company | Indication | 2028e SBI ($m) | Outcome |
| Zejula | GSK | Second-line ovarian cancer maintenance | 1,034* | Cancelled (FDA has asked GSK to restrict use to Brca +ve patients) |
| Xphozah (tenapanor) | Ardelyx | Control of serum phosphorus levels in adults with chronic kidney disease on dialysis | 357 | 9-4 in favour of monotherapy, 10-2 in favour of combo with phosphate binders |
| PT027 (albuterol/budesonide) | Astrazeneca/ Avillion | As-needed treatment or prevention of bronchoconstriction and for the prevention of exacerbations in patients with asthma 4 years of age and older | - | Favourable vote for those aged 18 and older but against in ages 4-11, and 12-17 years |
| Sabizabulin | Veru | Treatment of SARS-CoV-2 infection in moderate to severe Covid-19 infections at high risk of acute respiratory distress syndrome |
- | 5-8 against |
| *Forecasts not split by treatment line, restriction does not apply to Zejula's 1L maintenance use. Source: Evaluate Pharma, company releases, FDA adcom calendar. | ||||
| Supplementary and other notable approval decisions in November | |||
|---|---|---|---|
| Product | Company | Indication (clinical trial) | Outcome |
| Brexafemme | Scynexis/ Merck & Co | Recurrent vulvovaginal candidiasis (Candle) | Approved |
| Libtayo | Regeneron | 1L NSCLC (+chemo; Empower-Lung 3) | Approved |
| Vemlidy | Gilead | Chronic hepatitis B virus infection in paediatric patients 12 years of age and older with compensated liver disease (trial 1092) | Approved |
| Cotellic | Roche | Histiocytic neoplasms (Erdheim-Chester disease, Rosai-Dorfman disease, and Langerhans cell histiocytosis) (Ph2 NCT02649972) | Accelerated approval |
| Imfinzi + Imjudo + platinum-based chemo | Astrazeneca | Adult patients with stage IV NSCLC (Poseidon) | Approved |
| Adcetris | Seagen | Paediatric patients 2 years and older with previously untreated high risk classical Hodgkin lymphoma, (in combo with chemo) (AHOD1331) | Approved |
| Rylaze | Jazz | Additional dosing schedule, component of chemo regimen for ALL and lymphoblastic lymphoma patients who have developed hypersensitivity to E. coli-derived asparaginase | Approved |
| Rezvoglar (Lantus biosimilar) | Eli Lilly | Interchangeability designation (improve glycemic control in type 1 and type 2 diabetes) | Approved |
| Sezaby (phenobarbital sodium powder for injection) | Sun Pharma | Treatment of neonatal seizures (Neolev2) | Approved |
| Kineret | Swedish Orphan Biovitrum | Treatment of Covid-19 in hospitalised adults with pneumonia requiring supplemental oxygen who are at risk of progressing to severe respiratory failure | EUA |
| Bebtelovimab | Eli Lilly | Covid 19 omicron subvariants BQ.1 and BQ.1.1 showed a large reduction in susceptibility to the MAb | EUA revoked |
| Blenrep | GSK | Fourth-line relapsed or refractory multiple myeloma (accelerated approval) | Withdrawn from market (confirmatory Dreamm-3 failed) |
| Tecentriq | Roche | 1L urothelial bladder (accelerated approval) | Withdrawn from market (confirmatory Imvigor130 failed) |
| Source: Evaluate Pharma, company releases. | |||
