Global Regulatory Landscape

Abstract

There has been progress in ICH countries toward issuing regulatory guidance on development of drugs for certain types of MD. The EU is most advanced, with draft guidance for DMD and BMD issued in 2013, and a concept paper published in 2011. The U.S. seems to be taking a conservative approach, relying on current programs—such as Fast Track Designation, Breakthrough Therapy Designation, Accelerated Approval, and Priority Review—to help sponsors of MD therapies gain U.S. registration. In an unprecedented move, the FDA solicited the first-ever draft guidance for industry written on behalf of a patient advocacy group with a focus on DMD, although it is possible that the Agency may choose not to formally adopt the proposed guidance. Since there is no other official guidance in the US, the Parent Project Muscular Dystrophy (PPMD) document serves as a precedent for other types of muscular dystrophies. As of May 2015, there are no disease-modifying products approved for the treatment of MD, but that situation may soon change. In May 2014, reversing an earlier rejection, the EU Committee for Medicinal Products for Human Use (CHMP) recommended early (conditional) approval for PTC Therapeutics’ ataluren, a potential treatment for DMD. If the European Commission supports this decision, ataluren would be the first product approved, albeit conditionally, for MD—until the final Phase III data are available. This chapter provides an overview of the budding regulatory landscape for the treatment of MD in the EU and argues for more detailed FDA guidance.