Top Regulatory News Stories – Week Ending October 12, 2018

The US FDA gave Akcea Therapeutics and its parent company Ionis Pharmaceuticals the green light for Tegsedi (inotersen), a treatment for polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. Tegsedi was previously green-lit in Canada and the European Union.

The FDA has approved Leadiant Biosciences’ Revcovi (elapegademase-lvlr) for the treatment of adults and children with adenosine deaminase severe combined immune deficiency (ADA-SCID) — an ultra-rare, inherited genetic disorder.

The FDA has lifted the clinical hold placed in May this year and accepted the IND for CRISPR Therapeutics’ CTX001 for the treatment of sickle cell disease.

Eidos Therapeutics announced that the FDA has granted the company Orphan Drug Designation for AG10 for the treatment of transthyretin amyloidosis.

Synspira announced that it has been granted Orphan Designation by the FDA for poly (acetyl, arginyl) glucosamine (PAAG15A), for the treatment of cystic fibrosis.

Karyopharm Therapeutics announced that the FDA has accepted for filing with Priority Review its NDA seeking accelerated approval for selinexor, its first in class, oral SINE compound, as a new treatment for patients with penta-refractory multiple myeloma.

The FDA’s Oncologic Drugs Advisory Committee (ODAC) voted 16-0 that Celltrion’s and Teva’s CT-P10, a biosimilar to Roche’s Rituxan (rituximab), should be approved for three indications of the reference product.

In other news…

Ionis Pharmaceuticals and Roche have ink a deal worth more than $700 Million for dry AMD treatment collaboration:

Otsuka Pharmaceutical Co and Proteus Digital Health announced an expanded collaboration agreement to advance digital medicines for mental health:

Allogene’s IPO brought in $324 million as they plan to go into the clinic with their lead allogeneic CAR-T therapy for cancer next year:

Bristol-Myers Squibb’s cancer immunotherapy Opdivo failed to extend survival versus chemotherapy in previously treated patients with small-cell lung cancer (SCLC), a clinical setback that could weigh on the pharma’s chances of competing in the less common tumor type.


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