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Investors' top names, milestones and meetings in 4Q15

For investors who are not inclined to sit it out until the volatility abates, the fourth quarter is bursting with late-stage catalysts. There are no fewer than 17 PDUFA dates on the calendar before year end, and at least 11 Phase III data events.

The most eagerly anticipated event of 4Q15 - an FDA panel of Duchenne muscular dystrophy therapies - also is unconfirmed, as the agency still hasn't set a meeting date.

In addition to the bevy of regulatory and late-stage clinical milestones, investors agree the American Society of Hematology (ASH) meeting in December should provide much awaited visibility on durability of gene therapies and chimeric antigen receptor (CAR) T cell therapeutics.

Dialing up DMD

Investors who spoke to BioCentury are watching the reviews of BioMarin Pharmaceutical Inc. and Sarepta Therapeutics Inc. as an indicator of FDA's thinking on what constitutes sufficient evidence for approval in Orphan diseases beyond just DMD.

CEO Jean-Jacques Bienaimé has said BioMarin expects FDA to hold an advisory committee meeting this year for drisapersen. The antisense oligoribonucleotide that induces exon 51 skipping on the dystrophin gene has a Dec. 27 PDUFA date.

Sarepta has a Feb. 26 PDUFA date for its eteplirsen, a phosphorodiamidate morpholino oligomer (PMO) targeting exon 51. Numerous analysts and buysiders have speculated that an FDA panel likely would review both molecules together.

Deerfield's Jim Flynn said it will be interesting to see how FDA weighs drisapersen, which has a large database but a failed Phase III trial, and eteplirsen, which has fewer patients but a lot of patient advocate support.

Drisapersen missed the primary

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