The US FDA Grants Accelerated Approval Denali Therapeutics’ Avlayah (Tividenofusp alfa-eknm) for Hunter Syndrome (MPS II)

Shots: FDA has granted accelerated approval to Avlayah, an enzyme replacement therapy (ERT), for the treatment of presymptomatic or symptomatic MPS II pediatric pts (≥5kg), along with a RPD PRV to Denali; US availability is expected shortly Approval was based on the P-I/II trial of Avlayah (QW) in 47 pts, incl. ERT-naïve (n=15) & previously…

ByRidhi Rastogi2 days ago

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Dr. Reddy’s Reports the US FDA’s BLA Acceptance for DRL_AB (Biosimilar, Orencia)

Shots: The US FDA has accepted 351 (k) BLA for DRL_AB (IV), a proposed interchangeable biosimilar to Orencia (abatacept) Upon approval, DRL_AB will be indicated for adults with mod. to sev. active rheumatoid arthritis, adults with active psoriatic arthritis, & pts (≥6yrs.) with mod. to sev. active polyarticular juvenile idiopathic arthritis BLA was supported by extensive…

ByRidhi RastogiFebruary 23, 2026

NEWS

Summit Therapeutics Reports the US FDA’s BLA Acceptance for Ivonescimab to Treat EGFRm Non-Squamous NSCLC

Shots: The US FDA has accepted BLA of ivonescimab + CT for the treatment of EGFR-mutated locally advanced or metastatic non-squamous NSCLC post-TKI therapy (PDUFA: Nov 14, 2026) BLA was supported by P-III (HARMONi) trial assessing ivonescimab + Pt-doublet CT vs PBO + Pt-doublet CT in pts with EGFR-mutated, locally advanced or metastatic NSCLC who…

ByRidhi RastogiJanuary 30, 2026

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Summit Therapeutics Reports the US FDA’s BLA Submission for Ivonescimab to Treat EGFRm Non-Squamous NSCLC

Shots: The US FDA has received BLA of ivonescimab + CT for ≥2L treatment of pts with EGFR-mutated locally advanced or metastatic non-squamous NSCLC; decision is expected by Q4’26 BLA was supported by P-III (HARMONi) trial assessing ivonescimab + Pt-doublet CT vs PBO + Pt-doublet CT in pts with EGFR-mutated, locally advanced or metastatic NSCLC…

ByRidhi RastogiJanuary 13, 2026

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Annexon Seeks European Approval of Tanruprubart for Guillain-Barré Syndrome

Shots: The EMA has received MAA of tanruprubart for the treatment of Guillain-Barré syndrome (GBS) The MAA dossier demonstrated rapid neuroinflammation control & improved recovery, supported by a PoC study & a P-III trial against PBO, favorable risk/benefit, robust US & Southeast Asian biomarker data, PK (US/EU/SE Asia), & RWE matching P-III outcomes to IVIg…

ByRidhi RastogiJanuary 9, 2026

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Denali Therapeutics Announces Publishing of P-I/II Study of Tividenofusp Alfa (DNL310) in NEJM for Hunter Syndrome (MPS II)

Shots: The NEJM published results from the P-I/II study of Denali Therapeutics’ tividenofusp alfa (DNL310) for the treatment of Hunter syndrome (MPS II) The study evaluated treatment in 47 ERT-naïve (n=15) and previously treated (n=32) study participants (aged 0.3–13 [median, 5] years) demonstrating reduced levels of mean CSF levels of HS, mean urine HS levels,…

ByDipanshu DixitJanuary 2, 2026

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Ultragenyx Completes FDA Rolling BLA Submission for DTX401 in Glycogen Storage Disease Type Ia (GSDIa)

Shots: Ultragenyx has completed rolling submission of a BLA to the FDA seeking approval of DTX401 (pariglasgene brecaparvovec) for the treatment of GSDIa BLA is supported by data from 52 pts with 6yrs. of follow-up. Previously reported P-III (GlucoGene) trial results showed significant reductions in both quantity & frequency of daily cornstarch intake, improved euglycemia…

ByRidhi RastogiDecember 31, 2025

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INOVIO Reports the US FDA’s BLA Acceptance of INO-3107 for Recurrent Respiratory Papillomatosis (RRP)

Shots: The US FDA has accepted BLA of INO-3107 for the treatment for adults with RRP, granting PDUFA target action date of Oct 30, 2026 INOVIO filed a BLA for INO-3107 under the accelerated approval pathway & plans to engage with the FDA to align on requirements, while confirming it is not pursuing approval under…

ByRidhi RastogiDecember 30, 2025

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Zydus Partners with Bioeq for US Commercialization of Nufymco (Biosimilar, Lucentis)

Shots: Zydus Lifesciences has entered into a strategic licensing, supply & commercialization agreement with Bioeq for Nufymco (ranibizumab), an interchangeable biosimilar to Lucentis, for the US market, marking an expansion of Zydus’ US biosimilar portfolio Under the agreement, Bioeq will handle development, manufacturing, registration & supply of the finished product while Zydus will lead US…

ByPrince GiriDecember 25, 2025

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Kyverna Therapeutics Reports Topline P-II (KYSA-8) Trial Data of Miv-cel in Stiff Person Syndrome (SPS)

Shots: Kyverna has reported P-II (KYSA-8) trial data assessing a single dose of mivocabtagene autoleucel (miv-cel; KYV-101) in 26 SPS pts having an inadequate response with non-approved treatment options Trial met its 1EP with 46% median improvement in T25FW & 81% pts achieving a clinically meaningful ≥20% gain, plus significant benefits were seen across all…

ByRidhi RastogiDecember 17, 2025

The US FDA Grants Accelerated Approval Denali Therapeutics’ Avlayah (Tividenofusp alfa-eknm) for Hunter Syndrome (MPS II)

Dr. Reddy’s Reports the US FDA’s BLA Acceptance for DRL_AB (Biosimilar, Orencia)

Summit Therapeutics Reports the US FDA’s BLA Acceptance for Ivonescimab to Treat EGFRm Non-Squamous NSCLC

Summit Therapeutics Reports the US FDA’s BLA Submission for Ivonescimab to Treat EGFRm Non-Squamous NSCLC

Annexon Seeks European Approval of Tanruprubart for Guillain-Barré Syndrome

Denali Therapeutics Announces Publishing of P-I/II Study of Tividenofusp Alfa (DNL310) in NEJM for Hunter Syndrome (MPS II)

Ultragenyx Completes FDA Rolling BLA Submission for DTX401 in Glycogen Storage Disease Type Ia (GSDIa)

INOVIO Reports the US FDA’s BLA Acceptance of INO-3107 for Recurrent Respiratory Papillomatosis (RRP)

Zydus Partners with Bioeq for US Commercialization of Nufymco (Biosimilar, Lucentis)

Kyverna Therapeutics Reports Topline P-II (KYSA-8) Trial Data of Miv-cel in Stiff Person Syndrome (SPS)

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