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.
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.
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%.
Sarepta Therapeutics (SRPT) acquires a 26-acre multifunctional manufacturing facility in Massachusetts for ~$25M. It will use the site to make investigational exon skipping therapies for Duchenne muscular dystrophy. The transaction is expected to close in July.
The cGMP-qualified facility was built in 1996 and upgraded in 2006. It will support ~40 techs and support staff.
Based on updated guidance from the FDA regarding an early approval pathway for eteplirsen, Sarepta Therapeutics (SRPT) plans to file an NDA by the end of 2014.
The agency provided specific examples of additional safety and efficacy data for Duchenne muscular dystrophy that would enhance the acceptability of the NDA. The company will conduct several open label confirmatory studies later this year on patients with exon-51 amenable genotypes.
The company plans to conduct three studies: 1) ambulatory patients between the ages 7 and 16 years who can walk a minimum distance, 2) patients younger than 7 years, 3) DMD patients who cannot walk a minimum distance or who are non-ambulant.
The firm also plans to start a placebo-controlled study with one or more if its follow-on DMD exon-skipping drug candidates by year end.
Parent organizers of the grass roots campaign to get the regulator to approve the drug for children afflicted with Duchenne muscular dystrophy sent a letter to FDA Commissioner Margaret Hamburg today urging her to direct the agency to expedite its guidance to Sarepta Therapeutics (SRPT +4%) so it may file its NDA. The company has been waiting for said guidance for 16 months.
Once submitted, the organizers want an accelerated review of the application. Currently, there are no approved medications for the disease that afflicts 1 in 3,500 boys.
Parents of children suffering from Duchenne Muscular Dystrophy call for the FDA to accelerate its review of Sarepta Therapeutics' (SRPT) eteplirsen. Through their "The Race to Yes" campaign, they plan to petition the White House for a response to the issue. As of March 18, they have 43,750 of the required 100,000 signatures they need by the March 29 deadline.