PTC Therapeutics Wins on New DMD Agreement

Health and Healthcare

Published: July 7, 2016 12:40 pm

Last Updated: January 13, 2020 5:59 am

PTC Therapeutics Inc. (NASDAQ: PTCT) made waves on Thursday following a development in its nonsense mutation Duchenne muscular dystrophy (nmDMD) treatment. The company announced in conjunction with NHS England, that it has successfully negotiated a Managed Access Agreement (MAA) for Translarna (ataluren) for ambulatory patients aged five years and older with DMD.

This decision provides reimbursed patient access to Translarna in England via a five-year MAA. Translarna previously received a positive recommendation from the National Institute for Health and Care Excellence (NICE) in April of 2016, subject to PTC and NHS England finalizing the terms of the MAA. NICE is expected to issue final guidance later this month following execution of the MAA, with implementation soon after.

Primarily affecting males, Duchenne muscular dystrophy (DMD) is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of skeletal, diaphragm and heart muscles. Patients with DMD lose the ability to walk from as early as 10 years of age and experience life-threatening lung and heart complications in their late teens and early twenties.

Translarna received marketing authorization from the European Commission to treat DMD in August 2014, which is currently under annual review by the European Medicines Agency with an opinion on renewal expected mid-2016. Translarna is currently available to patients in over 20 countries through either expanded access programs or commercial sales.

Stuart W. Peltz, Ph.D., CEO of PTC, commented:

This is an important day in England for children and young adults suffering from DMD. We are extremely pleased to have reached a successful outcome with NHS England, which will provide long-awaited access to Translarna for patients with nonsense mutation DMD. We are grateful to the patients, families, advocacy groups and physicians for their tremendous effort in supporting PTC Therapeutics throughout this important and rigorous access process.

Shares of PTC were trading up 8% at $7.68 on Thursday, with a consensus analyst price target of $17.67 and a 52-week trading range of $5.27 to $62.15.

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