Italfarmaco Obtains the EC’s Conditional Approval for Duvyzat to Treat Duchenne Muscular Dystrophy (DMD)

Shots: The EC has granted conditional approval to Duvyzat (givinostat) for the treatment of ambulant DMD pts (≥6yrs.) on corticosteroids, regardless of underlying genetic mutation in all 30 EEA states Approval was based on the P-III (EPIDYS) study assessing Duvyzat (BID) vs PBO in ambulant DMD boys (n=179) on corticosteroid therapy Trial met its 1EP…

ByRidhi RastogiJune 9, 2025

News

Sarepta Therapeutics Reports the MHLW’s Conditional & Time-Limited Approval of Elevidys for Duchenne Muscular Dystrophy (DMD)

Shots: Japan's MHLW has granted conditional & time-limited approval to Elevidys for DMD pts (3–8yrs.) with no exon 8/9 deletions in the DMD gene & who are negative for anti-AAVrh74 Abs Approval was based on extensive clinical data, incl. 2yr. global P-III (EMBARK) trial showing motor function benefits vs external controls; Part 2 data was…

ByRidhi RastogiMay 14, 2025

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…

ByRidhi RastogiApril 28, 2025

News

Cumberland Pharmaceuticals Reveals Topline Data from P-II (FIGHT DMD) Trial of Ifetroban for Duchenne Muscular Dystrophy Heart Disease

Shots: Cumberland reported topline data from P-II (FIGHT DMD) trial assessing ifetroban (150/300mg, QD) vs PBO in DMD pts (n=41) for 12mos.; funded via the US FDA orphan products grant Study showed that ifetroban (300mg) led to 3.3% overall LVEF improvement (1EP), with a 1.8% increase vs 1.5% decline; on comparison with matched natural…

ByRidhi RastogiFebruary 5, 2025

News

Roche Reveals Data from P-III (EMBARK) Study of Elevidys in Ambulatory Boys with Duchenne Muscular Dystrophy

Shots: The P-III (EMBARK: N=126) study was carried out in 2 parts, with P1 involving ambulatory DMD boys (n=63; 4-7yrs.) to receive Elevidys (1.33x10^14 vg/kg) vs PBO while P2 involved crossing over of the patients (5-9yrs.); 59 crossed over to Elevidys post 52wks. Parts 1 (post 2yrs.) & 2 (post 1yr.) depicted improved NSAA…

ByRidhi RastogiJanuary 28, 2025

VIEWPOINTS

Enhancing DMD Care: Phil Lambert from Satellos in a Riveting Conversation with PharmaShots

Shots: Conventional therapeutic models for Duchenne Muscular Dystrophy (DMD) primarily focus on the role of dystrophin in maintaining muscle cell integrity. However, Satellos’ SAT-3247 takes a novel approach, targeting the lack of dystrophin in muscle stem cells to restore muscle fiber regeneration and repair Today at PharmaShots, we are joined by Phil Lambert, Chief Scientific…

BySaurabh ChaubeyNovember 20, 2024

INSIGHTS+

Disease of the Month – Duchenne Muscular Dystrophy (DMD)

Shots: The Disease of the Month report by PharmaShots aims to integrate an advanced approach to disease analysis. The report delves deep into the epidemiology, market size, disease management, available therapies, and key players involved For the April edition of the Disease of the Month report, PharmaShots brings an enlightening guide to Duchenne Muscular Dystrophy,…

BySaurabh ChaubeyApril 17, 2024

News

Italfarmaco’s Duvyzat Obtains the US FDA’s Approval to Treat Duchenne Muscular Dystrophy

Shots: The US FDA has granted approval to Italfarmaco’s Duvyzat (givinostat), histone deacetylase (HDAC) inhibitor, to treat Duchenne muscular dystrophy (DMD) patients (6yrs. or above) The approval was supported by the P-III (EPIDYS) study assessing the safety & efficacy of Duvyzat (BID) vs PBO in DMD ambulant male patients (n=179, 6yrs. or above) The trial…

ByDipanshu DixitMarch 22, 2024

INSIGHTS+

Insights+: EMA Marketing Authorization of New Drugs in December 2023

Shots: The EMA approved 3 BLA while 4 New Chemical Entities in December 2023, leading to treatments for patients and advances in the healthcare industry In December 2023, the major highlighted drugs were Jempreli to treat dMMR/MSI-H Primary Advanced or Recurrent Endometrial Cancer and Zilbrysq for the treatment of Generalized Myasthenia Gravis PharmaShots has compiled a list of a…

BySaurabh ChaubeyJanuary 23, 2024

INSIGHTS+

New Drug Designations – August 2023

Shots: PharmaShots' designation report provides a concise overview of several drugs and their designations by the US FDA, the EU, Japan, and China. This month’s report includes 8 biological drugs, 13 small molecules, 4 cell and gene therapies, 3 diagnostic tests and 5 devices Genprex’ Reqorsa, focused on the treatment of SCLC, is the drug to receive ODD…

BySenior EditorSeptember 18, 2023

Italfarmaco Obtains the EC’s Conditional Approval for Duvyzat to Treat Duchenne Muscular Dystrophy (DMD)

Sarepta Therapeutics Reports the MHLW’s Conditional & Time-Limited Approval of Elevidys for Duchenne Muscular Dystrophy (DMD)

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

Cumberland Pharmaceuticals Reveals Topline Data from P-II (FIGHT DMD) Trial of Ifetroban for Duchenne Muscular Dystrophy Heart Disease

Roche Reveals Data from P-III (EMBARK) Study of Elevidys in Ambulatory Boys with Duchenne Muscular Dystrophy

Enhancing DMD Care: Phil Lambert from Satellos in a Riveting Conversation with PharmaShots

Disease of the Month – Duchenne Muscular Dystrophy (DMD)

Italfarmaco’s Duvyzat Obtains the US FDA’s Approval to Treat Duchenne Muscular Dystrophy

Insights+: EMA Marketing Authorization of New Drugs in December 2023

New Drug Designations – August 2023

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