The FDA has ordered Novartis to halt enrollment and dosing in a set of trials for Zolgensma, their gene therapy for lethal cases of spinal muscular atrophy. And the latest setback triggered a frenzy of analyst notes as they sized up who among Novartis’ rivals in the field would benefit most.

The hold was implemented after Novartis’ subsidiary AveXis — still operating under the glare of an FDA investigation into an ethics scandal — informed regulators about concerns raised by an animal study pointing to “dorsal root ganglia (DRG) mononuclear cell inflammation, sometimes accompanied by neuronal cell body degeneration or loss.”

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What differentiates a woman leader in biopharma R&D from a man?

Not much, except there are fewer of them in senior posts. Data suggest women are not more risk-averse, family-oriented or less confident than their male counterparts — indeed the differences between the two sexes are negligible. But a glance at the top R&D positions in Big Pharma leaves little doubt that upward migration in the executive ranks of biopharma R&D is tough.

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An in-house FDA review of Amarin’s Vascepa raises a set of hurdles the biotech will have to clear if the biotech expects to get the long-awaited FDA approval that could set it on a path to superstar status. But it appears that Amarin has survived another potential setback without introducing a major new threat to its prospects.
The stakes don’t get much higher, with analysts saying a win this week for Amarin could lead to billions in new sales — provided the agency stamps it with an OK. And investors liked what they say in the FDA review this morning, bumping the stock $AMRN 17%.
The insider take at the agency includes a note on two new safety signals seen in the big cardio outcomes study of the omega-3 fatty acid drug that shocked many analysts with a solid set of efficacy data. There’s a key concern over whether the use of mineral oil in the placebo skewed LDL levels in such a way that tilted the data in Amarin’s favor.
The FDA overview was written by John Sharretts, the acting deputy director in the Division of Metabolism and Endocrinology Products. 
On the safety side, the internal review focused on a 3.1% versus 2.1% rate of adjudicated events of atrial fibrillation or atrial flutter requiring hospitalization. But they also say a-fib shouldn’t confound the benefit-safety of the drug — given the improvement on MACE — or prevent its use. And then there was also a higher rate of bleeding events in the drug arm.

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Solid Biosciences continues to be plagued by safety issues.

Close to 18 months after the gene therapy biotech was able to quickly shed an FDA hold on their lead Duchenne muscular dystrophy program for SGT-001, regulators have stepped back in to force another halt after another patient was hit hard by a set of serious adverse events remarkably similar to the first set.

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With Bristol-Myers Squibb’s Celgene buyout all but complete, the BD teams are working in perfect synchrony now. The Celgene side is going back to Skyhawk, a darling of the crowd that set out to drug RNA, and they’re adding a suite of new programs that mesh perfectly with the new regime in charge.

Celgene is shelling out $80 million in a cash upfront to add oncology, immuno-oncology and autoimmune diseases to the initial roundup of neurological targets mapped early in Skyhawk’s existence.

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