Duchenne Muscular Dystrophy
Marathon’s major misstep shows that drug pricing debate is here to stay
February 14, 2017Manufacturing 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 …
FDA approves first treatment for Duchenne muscular dystrophy
February 10, 2017Medical 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 …
Duchenne drug gets FDA approval in “unprecedented” ruling
September 20, 2016Manufacturing 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 …
FDA accepts Marathon’s Duchenne drug for priority review
August 11, 2016Research 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 …
NICE recommends PTC Therapeutics Duchenne muscular dystrophy treatment
July 20, 2016Research 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) …
Shares drop at Santhera after FDA requires more trial data DMD drug
July 15, 2016Medical 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) …
New FDA expanded access guidelines spell trouble for Sarepta
June 2, 2016Medical 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) …
Biomarin withdraws regulatory submissions for Duchenne muscular dystrophy drug
June 1, 2016Research 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), …
Shares at Sarepta Therapeutics jump after FDA delay in drug approval review
May 26, 2016Research 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 …
Nice extends price-negotiation talks for PTC’ muscle wasting drug
May 4, 2016Research 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 …
NICE recommends PTC Therapeutics’ Translarna to treat Duchenne muscular dystrophy
April 18, 2016Sales 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 …
PTC Therapeutics to cut jobs after FDA rejects Duchenne drug
March 24, 2016Manufacturing 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 …
US FDA sets April 25 date to review Sarepta’s DMD drug, shares jump
March 11, 2016Medical 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 …
FDA says no to BioMarin’s Duchenne Muscular Dystrophy drug
January 14, 2016Medical 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 …
The Pharmafocus debate: Can ultra-orphan drugs be made cheaper?
January 12, 2016Manufacturing 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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