Top Regulatory News Stories – Week Ending April 27, 2018

The FDA has approved Helsinn’s intravenous formulation of AKYNZEO® (a fixed antiemetic combination of fosnetupitant and palonosetron) as an alternative treatment option for patients experiencing Chemotherapy Induced Nausea and Vomiting.  Oral AKYNZEO® was previously approved by the FDA as a fixed combination oral agent in 2014.

Catalyst Pharmaceuticals announced its submission of a NDA to the FDA for Firdapse (amifampridine) for the symptomatic treatment of Lambert-Eaton myasthenic syndrome.

The EMA’s Committee for Orphan Medicinal Products issued a positive opinion recommending Alnylam’s early stage ALN-TTRsc02 for orphan status, with an official designation by the European Commission expected to follow.

Glenmark Pharmaceuticals has suspended a Phase II clinical trial at a site in India following media reports that villagers were allegedly “deceived” into participating.

Abeona Therapeutics Inc. announced that the EMA Committee for Orphan Medicinal Products has granted Orphan Drug Designation for Abeona’s gene therapy program ABO-202 for the treatment of subjects with neuronal ceroid lipofuscinosis, also known as Batten Disease

The FDA ordered a partial hold on Epizyme’s lead cancer drug tazemetostat following T-cell lymphoma case

Veloxis Pharmaceuticals announced today that the FDA has accepted their sNDA which seeks a new indication for ENVARSUS XR (tacrolimus extended-release tablets) for the prophylaxis of organ rejection in kidney transplant patients in combination with other immunosuppressants.

In the fifth version of FDA final guidance released Wednesday, the agency added a deadline for Type III drug master file submissions in eCTD format.

In a statement to the US Senate appropriations committee, FDA Commissioner Scott Gottlieb highlighted two issues that the agency would seek to address with additional funding:  rising drug prices and the need for real-time data:

Two more top FDA officials have retired marking the latest departures in a recent exodus of senior regulators from the agency.

In other news…

Takeda has upped its offer to buy Shire for a mix of stock and cash worth close to $65 billion, which is good enough for Shire to extend the deadline on their talks to May 8 after determining they were close to finalizing a pact.

Fresenius Kabi AG announced Monday it will terminate its $4.3 billion merger agreement with Akorn Inc., citing an internal investigation into practices at the generics maker, including what it contends were “material breaches of FDA data integrity requirements.”

UCB agreed to buy an NDA-ready epilepsy therapy from Proximagen in a $370 million deal.

Mylan Pharmaceuticals is laying off more than 400 employees at its Morgantown plant, according to a statement from the company

Nalproprion is buying all of Orexigen’s assets for $75 million, including worldwide rights to the weightloss drug Contrave (marketed under the brand name Mysimba in the EU).

The CEO of PixarBio, Frank Reynolds, was arrested and charged with securities fraud in a scheme to manipulate trading in the company’s shares:

Celldex Therapeutics is restructuring and cutting back in the aftermath of a disastrous Phase IIb readout. The company reported late Thursday that it is cutting 59 positions from its 2018 budgeted workforce, leaving it with 148 employees.

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