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on previous accelerated decisions continued in November, with Roche’s Tecentriq and GSK’s Blenrep and Zejula all being withdrawn in certain oncology indications after confirmatory studies failed.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.

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