Mon, Oct. 27, 9:13 AM| 1 Comment
Thu, Oct. 16, 3:37 PM
- Prosensa Holding N.V. (RNA +5.5%) commences its NDA submission process with the FDA for drisapersen for the treatment of Duchenne muscular dystrophy (DMD). The agency will review the application on a rolling basis as stipulated under Fast Track. It also designated drisapersen an Orphan Drug in June 2013.
- DMD-related tickers: (SRPT +7.1%)(PTCT +1.3%)(ROSG +8%)(OTCQB:MRNA)
Tue, Oct. 14, 12:44 PM
Wed, Aug. 13, 12:09 PM
Tue, Aug. 12, 5:42 PM
Mon, Aug. 4, 12:55 PM
- The EC grants conditional approval for PTC Therapeutics' (PTCT +1.3%) Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystophy (nmDMD) in ambulatory patients at least five years old.
- The conditional approval stipulates that PTC complete its Phase 3 clinical trial and submit additional safety and efficacy data from the trial.
- Related tickers: (SRPT -2.6%) (RNA +0.2%)
Mon, Jul. 7, 12:49 PM
Mon, Jul. 7, 12:44 PM
Thu, Jul. 3, 7:57 AM
- The FDA grants Fast Track designation to Akashi Therapeutics' lead product candidate HT-100 (delayed-release halofuginone) as a potential treatment to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with Duchenne Muscular Dystrophy (DMD). The agency has also designated HT-100 an Orphan Drug. The product candidate is currently being evaluated in a Phase 1b/2a clinical trial.
- Related tickers: (PTCT) (SRPT) (RNA)
Mon, Jun. 30, 8:31 AM
Thu, Jun. 5, 9:19 AM
- Analysts say that Shire's (SHPG) attractive pipeline and tax inversion-stoking location make it a prime acquisition target. For example, Allergan (AGN) approached the company in April about a potential deal. Its best defense against an unwanted takeover may be to go on the offensive and make an acquisition itself. This would seem to be a good strategy since CEO Flemming Ornskov likes to acquire. He has presided over at least six acquisitions since taking the helm.
- Cenkos Securities' Navid Malik perceives that Sarepta Therapeutics (SRPT), Prosensa (RNA) and ThromboGenics are attractive targets. So is NPS Pharmaceuticals (NPSP), but its CEO says the two firms aren't talking.
Tue, Jun. 3, 7:56 AM
- The FDA clarifies an accelerated approval pathway for Prosensa Holdings' (RNA) Duchenne Muscular Dystrophy drug drisapersen based on existing data. The company plans to file its NDA later this year and commits to two confirmatory post-approval studies.
- Prosensa will follow through on its plan to re-dose an initial cohort of boys in Q3 who have previously participated in drisapersen clinical studies.
- Shares are up 13% premarket on fair volume.
- Also benefiting from the good news is Sarepta Therapeutics' (SRPT) eteplirsen. The company also plans to file its NDA by year end.
Fri, May. 23, 12:45 PM
Fri, May. 23, 9:10 AM
Fri, May. 23, 8:41 AM
- PTC Therapeutics (PTCT) +119.2% premarket after the Committee for Medicinal Products for Human Use of the European Medicines Agency issues a positive opinion on PTCT's application for a conditional marketing authorization of its Ataluren muscular dystrophy treatment.
- The news is a major upside surprise: Credit Suisse says a potential early EU approval for Ataluren has not been priced in to shares.
- Others involved in Duchenne muscular dystrophy treatments also trade higher premarket: RNA +16%, SRPT +3%.
Tue, May. 20, 7:14 AM
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