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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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PTC Therapeutics
NEWS

PTC Therapeutics’ Sephience Receives the CHMP’s Positive Opinion for the Treatment of Phenylketonuria (PKU) 

Shots:  The CHMP has issued a positive opinion recommending marketing authorization for Sephience (sepiapterin) to treat PKU in patients of all ages and disease severities, expected the EC approval within two mos., with the decision applying across the EU including Iceland, Norway, and Liechtenstein  Sepiapterin's NDA is under FDA review (PDUFA: July 29, 2025), and…
April 25, 2025

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Akesobio
NEWS

Akeso’s Penpulimab-kcqx Receives the US FDA’s Approval for Nasopharyngeal Carcinoma (NPC)

Shots: The US FDA has approved penpulimab-kcqx + CT (cisplatin/carboplatin + gemcitabine) as 1L treatment for recurrent or metastatic non-keratinizing NPC in adults, & as monotx. for those with metastatic non-keratinizing NPC progressing on/after Pt-based CT & ≥1L prior therapy Approval was supported by 2 BLA submissions based on international P-III (AK105-304) trial & pivotal…
April 25, 2025

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Aclaris Therapeutics
NEWS

Aclaris Therapeutics Reports the US FDA’s IND Clearance of ATI-052 for Immuno-Inflammatory Diseases

Shots: The US FDA has granted IND clearance to ATI-052 for the initiation of P-Ia/Ib trial The P-Ia/Ib trial assessing single & multiple ascending doses of ATI-052 followed by PoC portion in an undisclosed indication, is expected to begin in Q2’25 ATI-052 blocks upstream TSLP receptor signaling & downstream IL-4/IL-13 activation to inhibit a central…
April 24, 2025

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Merck
NEWS

Merck’s Keytruda Regimen Receives Health Canada’s Approval for Malignant Pleural Mesothelioma (MPM)

Shots: Health Canada has approved Keytruda + pemetrexed + Pt-based CT as 1L treatment of unresectable advanced or metastatic MPM based on P-II/III (IND.227/KEYNOTE-483) trial led by CCTG in collaboration with NCIN & IFCT Trial assessed the regimen in MPM pts (n=440) who received either Keytruda (200mg, IV) + pemetrexed (500mg/m^2) & Pt-based CT on Day…
April 23, 2025

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