Vertex Pharmaceuticals’ Alyftrek Receives the CHMP’s Positive Opinion for the Treatment of Cystic Fibrosis

S R Shots: The CHMP has recommended Alyftrek (deutivacaftor/tezacaftor/vanzacaftor) to treat patients (≥6yrs.) with cystic fibrosis (CF), having at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Alyftrek is approved in the US and UK and is under review in Canada, Switzerland, Australia, and New Zealand Vanzacaftor &…

ByRidhi RastogiMay 1, 2025

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Satsuma Pharmaceuticals’ Atzumi (Dihydroergotamine) Nasal Powder Receives the US FDA’s Approval for Acute Treatment of Migraine

Shots: The US FDA has approved 505(b)(2) NDA of Atzumi (STS101) nasal powder for the acute treatment of migraine with or without aura in adults Approval was based on a P-I PK trial & P-III (ASCEND) trial, both showing fast absorption, rapid high DHE levels, & sustained DHE plasma concentrations over time Atzumi utilizes SMART…

ByRidhi RastogiMay 1, 2025

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Johnson & Johnson Reports the US FDA’s Approval of Imaavy (Nipocalimab-aahu) for Generalized Myasthenia Gravis (gMG)

Shots: The US FDA has approved Imaavy to treat antibody +ve gMG pts (≥12yrs.) under priority review based on the ongoing P-III (Vivacity-MG3) trial assessing Imaavy + SoC vs PBO + SoC in antibody +ve or -ve adults (N=199; 153 were Ab +ve); global regulatory review is ongoing Trial showed superior disease control per MG-ADL…

ByDipanshu DixitMay 1, 2025

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AbbVie’s Rinvoq (Upadacitinib) Receives the US FDA’s Approval for Giant Cell Arteritis

Shots: The US FDA has approved Rinvoq (15mg; QD) to treat adults with giant cell arteritis (GCA) following the EC’s Approval in Apr 2025 Approval was based on P-III (SELECT-GCA) trial assessing Rinvoq, where GCA pts (n=428) in 1st study period received either Rinvoq (7.5 or 15mg, QD) + 26wk. corticosteroid (CS) taper regimen or PBO +…

ByRidhi RastogiApril 30, 2025

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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,…

ByRidhi RastogiApril 30, 2025

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Regeneron’s Lynozyfic (Linvoseltamab) Receives the EC’s Conditional Approval for R/R Multiple Myeloma

Shots: The EC has granted conditional approval to Lynozyfic for the treatment of adults with r/r multiple myeloma based on P-I/II (LINKER-MM1) trial data assessing Lynozyfic in >300 pts; P-II dose expansion portion is ongoing. BLA under the US FDA’s Review (PDUFA: Jul 10, 2025) In the trial, 117 pts on 200mg had a 71% ORR, with…

ByRidhi RastogiApril 29, 2025

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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…

ByRidhi RastogiApril 29, 2025

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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…

ByRidhi RastogiApril 28, 2025

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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…

ByRidhi RastogiApril 28, 2025

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The CHMP Adopts Positive Opinion on Amgen’s Tepezza to Treat Thyroid Eye Disease

Shots: The EMA has recommended marketing authorization for Tepezza (teprotumumab) for adults with mod. to sev. thyroid eye disease The opinion was based on three randomized, PBO-controlled studies in 225 active and 62 chronic TED pts. After 24 wks., Tepezza significantly reduced eye protrusion (−2 to −2.3 mm) in protrusion of the eyeball from the…

ByDipanshu DixitApril 25, 2025

Vertex Pharmaceuticals’ Alyftrek Receives the CHMP’s Positive Opinion for the Treatment of Cystic Fibrosis

Satsuma Pharmaceuticals’ Atzumi (Dihydroergotamine) Nasal Powder Receives the US FDA’s Approval for Acute Treatment of Migraine

Johnson & Johnson Reports the US FDA’s Approval of Imaavy (Nipocalimab-aahu) for Generalized Myasthenia Gravis (gMG)

AbbVie’s Rinvoq (Upadacitinib) Receives the US FDA’s Approval for Giant Cell Arteritis

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

Regeneron’s Lynozyfic (Linvoseltamab) Receives the EC’s Conditional Approval for R/R Multiple Myeloma

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

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

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

The CHMP Adopts Positive Opinion on Amgen’s Tepezza to Treat Thyroid Eye Disease

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