FDA Posts Draft Guidance for DMD Treatment; Positive for BioMarin (BMRN), Says RBC

BioMarin Pharma (NASDAQ: BMRN) and Sarepta Therapeutics (NASDAQ: SRPT) were in focus Tuesday after the FDA issued draft guidance related to developing drugs for treatment of Duchenne Muscular Dystrophy and related dystrophinopathies (link to FDA Document).

In the view of analyst Michael Yee of RBC Capital, documents continues to support his buy thesis on BioMarin Pharma and probability of drisapersen approval.

"In guidance document we see a few comments that support BMRN's rationale for why drisapersen could still get approved," said Yee.

1. "'FDA has long stressed, however, that it is appropriate to exercise flexibility in applying the statutory standards to drugs for serious diseases with important unmet needs, while preserving appropriate guarantees for safety and effectiveness...' This reminds/stresses the view FDA may be understanding the need to be flexible (subjective) given the unmet need for DMD pts."
2. "'To support marketing approval, drug safety must be supported by an adequate number and duration of patient exposures to characterize drug risks. FDA generally will consider the serious and life-threatening nature of DMD ...when determining the minimum number and duration of patient exposures needed...' BMRN has large dataset and Phase III and Phase II package and good safety database with sufficient exposures to weigh risk/benefit (safety), in our view."
3. "'There is a strong rationale for treatment of patients at an early age because drugs that preserve muscle, in particular, may have the greatest effect on prognosis before muscle health has deteriorated.' - Generally supports BMRN view that Phase III was more difficult to show + data because enrolled sicker, more severe condition pts."

Yee pointed out two additional comments, including comments related to using 6-minute walk data in patients as young as 5 or 6 years old, and using more than one baseline assessment.

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Shares of BioMarin Pharmaceutical Inc. closed at $123.90 yesterday.

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