The US FDA Approves Ultragenyx’s Fayuvi for Sanfilippo Syndrome Type A

Shots: The FDA has granted full approval to Fayuvi (rebisufligene etisparvovec-hopf) for pediatric pts with Sanfilippo syndrome Type A, plus a Priority Review Voucher; commercial product will be available for shipment to QTCs within 30-60 days Approval was supported by pivotal Transpher A & long-term follow-up data, where Fayuvi-treated pts from the mITT population (n=17)…

ByRidhi RastogiSeptember 18, 2026

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Nanoscope Therapeutics Reports the US FDA BLA Acceptance of Mogenry for Retinitis Pigmentosa

Shots: The US FDA has accepted BLA of Mogenry (sonpiretigene isteparvovec, MCO-010), an optogenetic gene therapy designed for vision restoration for pts with retinitis pigmentosa (RP) having severe vision loss BLA was supported by the P-I/IIa trial, & the P-IIb/III (RESTORE) trial, where RESTORE met its 1 & 2EPs, showing improvements in visual acuity at…

ByRidhi RastogiSeptember 9, 2026

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AstraZeneca Reports P-III OBERON and TITANIA Results for Tozorakimab in COPD at ERS’26

Shots: AstraZeneca reported full results from the P-III OBERON and TITANIA studies, showing that tozorakimab 300 mg Q4W reduced moderate-to-severe COPD exacerbations versus PBO in pts. receiving inhaled standard of care In the primary population of former smokers, tozorakimab reduced exacerbations by 29% in OBERON and 34% in TITANIA, while reductions in the overall population…

ByPrince GiriSeptember 8, 2026

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uniQure Reports the US FDA BLA Submission for Ifezuntirgene Inilparvovec to Treat Huntington’s Disease

Shots: The US FDA has received a BLA for the accelerated approval of ifezuntirgene inilparvovec (AMT-130) for the treatment of Huntington’s disease, plus a request for Priority Review BLA was supported by the 3yr. data analysis from the P-I/II trial of AMT-130 vs a propensity score-matched external control derived from the Enroll-HD natural history database;…

ByRidhi RastogiSeptember 2, 2026

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Alvotech Reports US FDA BLA Acceptance for AVT80 (Biosimilar, Entyvio)

Shots: The US FDA has accepted a BLA for AVT80, a proposed interchangeable biosimilar to Entyvio (vedolizumab) PFS & autoinjector for SC administration BLA was backed by analytical, PK & immunogenicity data, showing biosimilarity with the reference product. Plus, under a partnership with Teva, Alvotech is responsible for development & manufacturing of AVT80, while Teva…

ByRidhi RastogiAugust 31, 2026

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PTC Therapeutics to Acquire Sangamo Therapeutics’ ST-920 Gene Therapy Program

Shots: PTC Therapeutics has been selected as the winning bidder to acquire ST-920, a BLA-stage, one-time AAV gene therapy for Fabry disease, from Sangamo Therapeutics through a competitive bankruptcy auction As per the terms, PTC Therapeutics will pay $111M upfront, with up to $100M in contingent milestone payments tied to certain regulatory approvals; expected to…

ByRidhi RastogiAugust 13, 2026

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Fresenius Kabi Reports FDA and EMA Acceptance of PB016 (Biosimilar, Entyvio) for Review

Shots: The US FDA and EMA have accepted the BLA & MAA, respectively, for PB016, a biosimilar version of Entyvio (vedolizumab) lyophilized vial for IV administration BLA was supported by an extensive development program assessing PB016 vs Entyvio, to evaluate its similarity to the reference product, consistent with regulatory requirements for biosimilar approval PB016 is…

ByRidhi RastogiAugust 3, 2026

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Nippon Shinyaku Exercises US Option for AB2 Bio’s Tadekinig Alfa

Shots: Nippon has exercised its exclusive US commercialization option for tadekinig alfa in Primary Monogenic IL-18-Driven Hyperinflammatory Syndrome with NLRC4 & XIAP mutations, with AB2 Bio retaining US rights to all other indications & all rights outside the US AB2 Bio received a $6M option fee in 2025 & will now receive a $30M option…

ByRidhi RastogiJuly 30, 2026

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Hansoh Pharma Reports P-III (ARTEMIS-011) Trial Data on Ris-Rez for Osteosarcoma

Shots: Hansoh has reported P-III (ARTEMIS-011) trial data assessing risvutatug rezetecan (HS-20093), a B7-H3–directed ADC, vs CT in pts with osteosarcoma who had progressed or relapsed after receiving ≥2 prior lines of systemic therapy Trial met its 1EP of improved PFS as assessed by the IRC, with consistent benefits observed across 2EPs, incl. OS & investigator-assessed…

ByRidhi RastogiJuly 28, 2026

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Eli Lilly Reports P-III (TRIUMPH-2 and TRIUMPH-3) Results on Retatrutide for Obesity

Shots: TRIUMPH-2 assessed retatrutide (4, 9, or 12mg, QW) vs PBO in 1152 pts with T2D & obesity/overweight, while TRIUMPH-3 assessed retatrutide (9 or 12mg, QW) in 1949 pts with severe obesity & CV disease; trials used a stepwise dose-escalation approach at Q4W In TRIUMPH-3, 12mg reduced TGs, non-HDL cholesterol, systolic BP, waist circumference, &…

ByRidhi RastogiJuly 24, 2026

The US FDA Approves Ultragenyx’s Fayuvi for Sanfilippo Syndrome Type A

Nanoscope Therapeutics Reports the US FDA BLA Acceptance of Mogenry for Retinitis Pigmentosa

AstraZeneca Reports P-III OBERON and TITANIA Results for Tozorakimab in COPD at ERS’26

uniQure Reports the US FDA BLA Submission for Ifezuntirgene Inilparvovec to Treat Huntington’s Disease

Alvotech Reports US FDA BLA Acceptance for AVT80 (Biosimilar, Entyvio)

PTC Therapeutics to Acquire Sangamo Therapeutics’ ST-920 Gene Therapy Program

Fresenius Kabi Reports FDA and EMA Acceptance of PB016 (Biosimilar, Entyvio) for Review

Nippon Shinyaku Exercises US Option for AB2 Bio’s Tadekinig Alfa

Hansoh Pharma Reports P-III (ARTEMIS-011) Trial Data on Ris-Rez for Osteosarcoma

Eli Lilly Reports P-III (TRIUMPH-2 and TRIUMPH-3) Results on Retatrutide for Obesity

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