Sarepta may have launched its first therapy for Duchenne muscular dystrophy in the US, but it is facing potential competition from a plethora of late-stage DMD therapies; it is backing research that could lead to new gene therapies able to treat most affected patients, rather than a subgroup of individuals with a particular gene mutation.

Several companies have DMD drug candidates waiting in the wings to follow Sarepta’s Exondys 51 into the commercial market after FDA approval of the exon-skipping therapy, which could be the backbone for future combination treatment regimens.

Derived from Strategic Transactions, Informa’s premium source for tracking life sciences deal activity, the Dealmaking column is a survey of recent health care transactions listed by relevant industry segment – In Vitro Diagnostics, Medical Devices, and Pharmaceuticals – and then categorized by type – Acquisition, Alliance, or Financing. This month’s column covers deals announced August-September 2014.

The economics of orphan drugs have been pretty straightforward: Transformative treatments for rare diseases deserve premium prices. And so far, payors – at least the larger ones – haven’t really balked at those price tags. But stakeholders are starting to ask whether the orphan drug pricing benchmark set by Ceredase 20 years ago is right for all rare disease drugs. Industry needs to have good answers.