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)
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/
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/
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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