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Duchenne Muscular Dystrophy

Showing 15 posts of 34 posts found.

marathon_logo

Marathon’s major misstep shows that drug pricing debate is here to stay

February 14, 2017
Manufacturing and Production, Sales and Marketing Duchenne Muscular Dystrophy, Emflaza, marathon

Marathon Pharmaceuticals’ approval, last week, by the FDA for its treatment for Duchenne muscular dystrophy would usually have been cause …

News
fda2outsideweb

FDA approves first treatment for Duchenne muscular dystrophy

February 10, 2017
Medical Communications, Sales and Marketing Duchenne Muscular Dystrophy, FDA, marathon, marathon pharmaceuticals

The FDA has announced that it has approved Emflaza (deflazacort), developed by Marathon Pharmaceuticals. The treatment will arrive on the …

News

Duchenne drug gets FDA approval in “unprecedented” ruling

September 20, 2016
Manufacturing and Production, Sales and Marketing Duchenne Muscular Dystrophy, FDA, sarepta

The FDA has gone against the advice of its own staff to approve the first treatment of Duchenne muscular dystrophy …

News

FDA accepts Marathon’s Duchenne drug for priority review

August 11, 2016
Research and Development, Sales and Marketing Duchenne Muscular Dystrophy, FDA, deflazacort, marathon, marathon pharmaceuticals

Marathon Pharmaceuticals has announced that the US Food and Drug Administration (FDA) has accepted and granted a priority review for …

News
lab

NICE recommends PTC Therapeutics Duchenne muscular dystrophy treatment

July 20, 2016
Research and Development, Sales and Marketing Duchenne Muscular Dystrophy, PTC Therapeutics

PTC Therapeutics has announced that the National Institute for Health and Care Excellence has issued final guidance recommending Translarna (ataluren) …

News
lab

Shares drop at Santhera after FDA requires more trial data DMD drug

July 15, 2016
Medical Communications, Research and Development Duchenne Muscular Dystrophy, FDA, santhera

Santhera Pharmaceuticals (SIX: SANN) has seen shares fall as much as 38% after the US Food and Drug Administration (FDA) …

News

New FDA expanded access guidelines spell trouble for Sarepta

June 2, 2016
Medical Communications, Research and Development, Sales and Marketing Duchenne Muscular Dystrophy, Eteplirsen, FDA, compassionate grounds, sarepta

New guidelines intended to streamline the process of expanded access to medicines by the US Food and Drug Administration (FDA) …

News

Biomarin withdraws regulatory submissions for Duchenne muscular dystrophy drug

June 1, 2016
Research and Development, Sales and Marketing BioMarin, Duchenne Muscular Dystrophy, EMA, FDA, Kyndrisa, failure, rejection

Biomarin (NASDAQ: BMRN) has announced that it has withdrawn its marketing authorisation application for Duchenne muscular dystrophy drug, Kyndrisa (drisapersen), …

News
lab

Shares at Sarepta Therapeutics jump after FDA delay in drug approval review

May 26, 2016
Research and Development, Sales and Marketing Duchenne Muscular Dystrophy, FDA, Sarepta Therapeutics, approval, delay, delayed announcement, muscular dystrophy, sarepta, treatment

Sarepta Therapeutics (NASDAQ: SRPT) has witnessed a sharp rise in the price of its shares after the US Food and …

News

Nice extends price-negotiation talks for PTC’ muscle wasting drug

May 4, 2016
Research and Development, Sales and Marketing Duchenne Muscular Dystrophy, NICE, PTC Therapeutics, Translarna, market access, regulation

UK regulators have extended the cost negotiation talks with PTC Therapeutics (Nasdaq: PTCT) by a week for the drug to …

News
drugs1

NICE recommends PTC Therapeutics’ Translarna to treat Duchenne muscular dystrophy

April 18, 2016
Sales and Marketing DMD, Duchenne Muscular Dystrophy, NICE, PTC Therapeutics, Translarna, regulation

Shares in drug maker PTC Therapeutics (Nasdaq: PTCT) jumped after the National Institute for Health and Care Excellence (NICE) backed …

News
ptc_therapeutics_logo

PTC Therapeutics to cut jobs after FDA rejects Duchenne drug

March 24, 2016
Manufacturing and Production Duchenne Muscular Dystrophy, PTC Therapeutics

PTC Therapeutics has announced plans to reduce its operating expenses that will see it cut almost a fifth of its …

News
shutterstock_38078521

US FDA sets April 25 date to review Sarepta’s DMD drug, shares jump

March 11, 2016
Medical Communications, Research and Development Duchenne Muscular Dystrophy, Eteplirsen, Sarepta Therapeutics, US FDA

Shares in US-based Sarepta Therapeutics (Nasdaq: SRPT) jumped nearly 15% Thursday after the US Food and Drug Administration (FDA) said it …

News
FDA

FDA says no to BioMarin’s Duchenne Muscular Dystrophy drug

January 14, 2016
Medical Communications, Sales and Marketing BioMarin, Duchenne Muscular Dystrophy, FDA, Kyndrisa, drisapersen

The FDA has rejected BioMarin’s Duchenne Muscular Dystrophy (DMD) drug Kyndrisa, saying there was not enough evidence that the drug …

News
DMD

The Pharmafocus debate: Can ultra-orphan drugs be made cheaper?

January 12, 2016
Manufacturing and Production, Research and Development, Sales and Marketing Drug pricing, Duchenne Muscular Dystrophy, Translarne, access to medicines, ataluren, muscular dystrophy, rare disease, ultra orphan drugs, ultra-orphan drugs

The recent reimbursement wrangle between PTC Therapeutics and NICE, the UK healthcare watchdog, has once again raised difficult questions about …

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