Regulatory Roundup: Top News Stories – Week Ending August 11, 2017

There have not been any new drug approvals so far this week but the FDA granted a few new Orphan Drug Designations and Priority Reviews. Here is my summary of the top regulatory news stories this week for pharma and biotech:

The FDA is on track for most generic drug approvals ever for Fiscal Year 2017. In the first 10 months of FY2017 the FDA has approved 633 generic drugs, compared with a total of 651 approvals in all of FY 2016.

The FDA said that it is illegal to perform research on mitochondrial replacement technology (MRT) since it would involve the intentional creation of a genetically modified embryo:

The FDA also finalized guidance to formalize a program that qualifies medical device development tools (MDDT) for use in device development and evaluation programs.

FDA also drafted guidance on post-approval manufacturing changes for biologics

FDA granted Orphan Drug Designation to Omeros’ OMS721 for the treatment of Immunoglobulin A (IgA) Nephropathy

Bluebird Bio has entered into talks with UK regulators regarding a possible launch of its gene therapies for rare diseases:

The FDA has accepted Genentech’s sNDA and granted Priority Review for Zelboraf (vemurafenib) for Erdheim-Chester disease (a rare, serious blood disease characterized by the abnormal multiplication of certain white blood cells called histiocytes)

Capricor Therapeutics announced that the FDA has granted Rare Pediatric Disease Designation to CAP-1002, Capricor’s development candidate for the treatment of Duchenne muscular dystrophy:

Tamir Biotechnology announced that ranpirnase has received Orphan Drug Designation from the US FDA for the treatment of Ebola Virus Disease.

Ocugen, Inc. announced that the FDA has granted Orphan Drug Designation for OCU300 (brimonidine tartrate) for the treatment of ocular graft versus host disease (oGVHD).

The FDA has granted priority review for Gilead Sciences’ NDA for its investigational, fixed-dose combination of bictegravir and emtricitabine/tenofovir alafenamide for the treatment of HIV-1 infection.

The FDA will not be scheduling an Advisory Committee meeting for Kite Pharma’sclosely-watched CAR-T drug axi-cel. Kite faces a November 29 deadline for its marketing decision from the FDA:

In other news…

German dialysis giant Fresenius is paying $2 billion to acquire NxStage Medical, a Lawrence, MA-based developer of products for patients dealing with kidney failure.

Eli Lilly’s migraine drug Lasmiditan has met its primary endpoint in a second phase 3 trial.

Zynerba Pharmaceuticals has reported that its cannabis-based epilepsy gel ZYN002 (cannabidiol gel) has failed to meet the primary point in a phase 2 clinical trial.

Contract research organization PRA Health Sciences is purchasing health data and analytics expert Symphony Health for $530 million. Symphony provides insights that span across market prediction, physician prescribing, payer reimbursement and patient treatment patterns.

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