August was an excellent month for biopharma in terms of approvals vs. CRLs, with 8 of 10 therapeutic candidates with PDUFA dates in August 2024 getting approved by the U.S. FDA! In addition, 1 PDUFA date was extended and we only identified 1 CRL (i.e. rejection). Learn more below about each of the therapeutics and new indications receiving the green light from the US FDA.
FIG 1. August PDUFA Calendar
Zevra AdComm for Arimoclomol for Niemann-Pick disease type C
AdComm voted 11-5 in favor of approving arimoclomol
Agenda: Resubmitted NDA
FDA docs posted 7/31 & highlight concerns on...
» Validity of revised primary endpoint (R4DNPCCSS)
» Uncertainty about actual benefit based on the revised data
» Whether additional data adequately supports efficacy
» Overall strength of evidence
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Adaptimmune PDUFA for Tecelra® for Synovial Sarcoma - APPROVED
» First approved engineered cell therapy for solid tumor
» For adults w/ advanced synovial sarcoma who have undergone prior chemo & meet specific genetic criteria (HLA and MAGE-A4)
» Accelerated Approval
» Based on initial response rates
» Further studies required to confirm long-term benefits
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Citius PDUFA for Lymphir™ for r/r Cutaneous T-Cell Lymphoma - APPROVED
» 1st systemic Tx targeting IL-2 receptor on malignant T-cells & Tregs
» Expected launch w/in 5 months
» 1st novel targeted systemic therapy approved for r/r CTCL since 2018
» Based on Ph3 Pivotal Study 302
» Showed 36% ORR, reduced skin disease, improved pruritis, & no cumulative toxicity
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Gilead PDUFA for Livdelzi® for Primary Biliary Cholangitis - APPROVED
» First treatment to significantly reduce key biomarkers, normalize ALP, & alleviate itching vs placebo
» Accelerated approval based on ALP reduction
» Further trial/s required to confirm clinical benefit
» Not recommended for patients w/ decompensated cirrhosis
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Ascendis PDUFA for Yorvipath® for Hypoparathyroidism in Adults - APPROVED
» The first & only Tx approved for hypoparathyroidism
» Once-daily prodrug of parathyroid hormone
» Hypoparathyroidism affects estimated 70k to 90k people in US
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Servier/Agios PDUFA for Voranigo® for Grade 2 IDH-mutant glioma - APPROVED
» 1st FDA-approved targeted therapy for Grade 2 IDH-mutant glioma
» Positive safety profile in Ph3 INDIGO trial
» Significant improvement in progression-free survival
» Agios receives $200M from Servier related to 2021 oncology business divestiture
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Regeneron PDUFA for Linvoseltamab BLA for R/R Multiple Myeloma - CRL
» Linvoseltamab for R/R multiple myeloma
» CRL due to findings from a pre-approval inspection at 3rd party fill/finish manufacturer 🏭
» FDA reinspection in the coming months
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GSK PDUFA for Jemperli Expanded use for Endometrial Cancer - APPROVED
» Jemperli + chemotherapy
» Now approved for all adult patients w/ primary advanced or recurrent endometrial cancer
» Now includes MMRp/MSS tumors (majority of endometrial cancer cases)
» Showed 31% reduction in risk of death vs chemo alone
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Vericel PDUFA for Arthroscopic Delivery of MACI Arthro - APPROVED
» Restorative biologic cartilage repair product
» Expands MACI® label for arthroscopic administration
» Targets largest segment of MACI's $3B addressable market
» Less invasive vs current approach
» Uses custom-designed instruments for delivery
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JNJ PDUFA for Rybrevant® plus Lazcluze™ for NSCLC - APPROVED
» 1st chemo-free regimen for NSCLC
» First-line treatment of EGFR-mutated advanced lung cancer
» Ph3 MARIPOSA study
→ Superior progression-free survival vs osimertinib
→ Reduced disease progression risk or death by 30%
→ Extended median duration of response by 9 months
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ARS Pharma PDUFA for neffy® for Type 1 Allergic Reactions - APPROVED
» The first needle-free treatment for Type I allergic reactions, including anaphylaxis
» New delivery method for epinephrine in over 35 years
» For adults & children weighing at least 30 kg
» ARS Pharma is well-capitalized to launch & commercialize neffy® in US
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Novartis PDUFA for Fabhalta® New Indication - APPROVED
» 1st-in-class complement inhibitor for reducing proteinuria in primary IgA nephropathy
» FDA accelerated approval
» Ph3 APPLAUSE-IgAN study
» 44% proteinuria reduction vs placebo
» Addresses unmet need, up to 50% of IgAN patients progress to kidney failure
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Incyte and Syndax PDUFA for Niktimvo™ for Chronic GVHD - APPROVED
» For adults & children weighing at least 40 kg after failure of 2 prior systemic therapies
» 1st approved anti-CSF-1R antibody targeting inflammation & fibrosis in chronic GVHD
» AGAVE-201 study: durable responses across all organs & patient subgroups
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SC Pharma PDUFA for Furoscix® sNDA - APPROVED
» Indication expanded to treat congestion due to fluid overload in adult patients w/ chronic heart failure, regardless of NYHA functional class
» Can now be used in NYHA Class IV chronic heart failure patients
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Article History:
MV/JD/DG (9/4/24)
Not legal, investing, or tax advice.