US FDA approval and panel tracker: April 2023
Biogen and Ionis’s Qalsody is already backed by a positive panel, and received a US greenlight last month in ALS patients with Sod1 mutations. The accelerated decision was based on a neurofilament biomarker, and the ongoing Atlas trial in pre-symptomatic carriers will serve as the confirmatory study. Stifel analysts highlighted the broader readthrough to the neurology division's openness to biomarkers, noting Sarepta’s closely watched panel this month for the Duchenne muscular dystrophy gene therapy SRP-9001. Elsewhere manufacturing issues scuppered Ascendis’s TransCon PTH, intended to treat hypoparathyroidism, and Lilly’s mirikizumab in ulcerative colitis. In addition, a panel to discuss Lynparza’s use in first-line prostate cancer voted to restrict the label to Brca-positive patients, based on data from the Propel study. Attention now turns to the EU, where the EMA gave Lynparza a broad prostate cancer label at the end of last year.
| Notable first-time US approval decisions in April 2023 | ||||
|---|---|---|---|---|
| Project | Company | Indication(s) | 2028e SBI ($m) | Outcome |
| TransCon PTH | Ascendis | Adults with hypoparathyroidism | 1,214 | Deficiencies identified in NDA, then CRL (manufacturing issues) |
| Mirikizumab (Omvoh) | Lilly | Ulcerative colitis | 630 | CRL (manufacturing issues) |
| LetibotulinumtoxinA/ Botulax/ Letybo |
Hugel | Glabellar lines | 276 | CRL (second, inspection issues) |
| Quizartinib (Vanflyta) + standard cytarabine and anthracycline induction | Daiichi Sankyo | Newly diagnosed FLT3-ITD +ve AML | 120 | Extended to 24 July owing to REMS |
| Omisirge (omidubicel) | Gamida Cell | Cell therapy for patients with blood cancers to reduce risk of infection following stem cell transplantation | 82 | Approved (extended Pdufa had been expected 1 May) |
| Qalsody (tofersen) | Biogen/Ionis | ALS associated with a mutation in Sod1 gene | 10 | Approved (accelerated) |
| Vowst (SER-109) | Seres | Clostridioides difficile infection | 5 | Approved |
| Abilify Asimtufii (Aripiprazole 2-month) | Otsuka/ Lundbeck |
Treatment of schizophrenia and maintenance treatment of bipolar I disorder | - | Approved |
| SBI: sales by indication. Sources: Evaluate Pharma & company releases. | ||||
| Advisory committee meetings in April 2023 | ||||
|---|---|---|---|---|
| Project | Company | Indication | 2028e SBI ($m) | Outcome |
| Lynparza + Zytiga + prednisone/prednisolone | Astrazeneca/Merck & Co | 1st-line castration-resistant prostate cancer (Propel) | 1,339* | 11-1 vote to restrict label to Brca+ve patients (Pdufa had been expected in March) |
| Rexulti | Otsuka/ Lundbeck |
Agitation associated with Alzheimer’s dementia | 783 | 9-1 in favour (Pdufa 10 May) |
| Sulbactam-durlobactam for injection (Sul-Dur) | Innoviva (Entasis) | Hospital‐acquired and ventilator‐associated bacterial pneumonia caused by Acinetobacter baumannii‐calcoaceticus complex in adults | 82 | 12-0 in favour (Pdufa 29 May) |
| *Already on the market in different treatment line. Sources: Evaluate Pharma, FDA adcom calendar & company releases. | ||||
| Supplementary and other notable approval decisions in April 2023 | |||
|---|---|---|---|
| Product | Company | Indication (clinical trial) | Outcome |
| Polivy + Rituxan + R-CHP | Roche | Previously untreated DLBCL (Polarix) | Approved |
| Padcev + Keytruda | Seagen/Astellas (Pfizer) | 1L urothelial cancer cisplatin ineligible patients (Ph1b/2 EV-103/Keynote-869 dose escalation/cohort A & cohort K) | Approved (accelerated) |
| Trikafta | Vertex | Cystic fibrosis in children aged 2-5 | Approved |
| Prevnar20 | Pfizer | Pneumococcal vaccine in infants & children 6 weeks to 17 years old (ph3 NCT04382326, NCT04546425, NCT04379713, Ph2) | Approved |
| Qulipta | Abbvie | Preventative treatment of chronic migraine (Progress) | Approved |
| Sogroya | Novo Nordisk | Growth hormone deficiency in children up to 11 years old (Real4) | Approved |
| Hyqvia | Takeda | Primary immunodeficiency in children aged 2-16 | Approved |
| Tepezza | Horizon | Thyroid eye disease regardless of thyroid eye disease activity or duration (ph4 NCT04583735) | Approved |
| Rizafilm Versafilm (Rizaport) | Intelgenx/Gensco | Acute migraine | Approved |
| mdc-IRM (Uzedy) (risperidone extended-release) | Teva/Medincell | Maintenance treatment of schizophrenia | Approved |
| Lumryz (FT218) | Avadel | Treatment of excessive daytime sleepiness or cataplexy in adults with narcolepsy | Final approval |
| Gohibic (vilobelimab) | Inflarx | Treatment of critically ill adult Covid-19 patients (Panamo) | EUA granted |
| AVT02 | Alvotech/Teva | Humira biosimilar interchangeability | CRL (second, for manufacturing issues) |
| Imbruvica | Abbvie/J&J | Previously untreated mantle cell lymphoma (Shine) and relapsed or refractory marginal zone lymphoma (Selene) | Withdrawn, confirmatory data insufficient to support conversion to full approval (Shine did not show OS; Selene did not meet PFS) |
| Makena (and generics) | Covis (private) | Reducing risk of preterm birth in women who have a history of singleton spontaneous preterm birth | Withdrawn (final decision) |
| Monovalent mRNA Covid-19 vaccines | Moderna and Pfizer/Biontech | Immunisation to prevent Covid-19 | EUA removed |
| Sources: Evaluate Pharma & company releases. | |||
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