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Abeona Therapeutics
NEWS

Abeona Therapeutics’ Zevaskyn Receives the US FDA Approval for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Shots: The US FDA has approved Zevaskyn (prademagene zamikeracel) for treating wounds in adult & pediatric RDEB pts based on the intra-pts controlled P-III (VIITAL) trial & has also granted RPD PRV to Abeona, which the company intends to monetize; Zevaskyn to be commercially available in Q3’25 The P-III Trial assessed Zevaskyn in RDEB pts,…
April 30, 2025

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NEWS

The CHMP Adopts Positive Opinion on Alnylam Pharmaceuticals’ Vutrisiran to Treat ATTR Amyloidosis with Cardiomyopathy (ATTR-CM)

Shots: The CHMP has recommended vutrisiran to treat ATTR-CM adults based on global P-III (HELIOS-B) study assessing it vs PBO in ATTR-CM pts; EC's decision expected in Jun 2025. MAA under PMDA review, with global applications planned in 2025 & beyond Study met all ten 1 & 2EPs across both overall & monotx. arms, showing improved…
April 29, 2025

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Italfarmaco
NEWS

Italfarmaco Obtains the CHMP’s Positive Opinion for Conditional Marketing of Duvyzat to Treat Duchenne Muscular Dystrophy (DMD)

Shots: Opinion for Duvyzat to treat ambulant DMD pts (≥6yrs.) with corticosteroids is based on P-III (EPIDYS) study assessing Duvyzat (BID) vs PBO in ambulant boys (n=179); EC’s decision expected in Jul 2025 Trial met its 1EP by improving four-stair climb completion time, with benefits seen in 2EPs  (NSAA & MRI fat infiltration) & a…
April 28, 2025

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Jazz Pharmaceuticals
NEWS

Jazz Pharmaceuticals’ Zanidatamab Secures the CHMP Positive Opinion to Treat HER2+ Biliary Tract Cancer (BTC)

Shots: The CHMP has recommended conditional marketing approval of zanidatamab for the treatment of inoperable locally advanced or metastatic HER2+ (IHC 3+) biliary tract cancer (BTC) adults, who were previously treated with ≥1L of therapy; EC’s potential approval will be valid in 30 EEA states Opinion was based on P-IIb (HERIZON-BTC-01) trial to assess anti-tumor…
April 28, 2025

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NEWS

Purpose Pharma’s Attrogy Receives the CHMP’s Positive Opinion for the Treatment of Hereditary Transthyretin-mediated Amyloidosis  

Shots:  The CHMP has recommended Attrogy (diflunisal) to treat hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult pts. with stage 1 or stage 2 polyneuropathy  The EC will make decision within 67 days, and if approved, Attrogy will be authorized in all EU & EEA countries Iceland, Lichtenstein and Norway  Diflunisal has received EU ODD for…
April 25, 2025

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