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A collection of recent regulatory approvals and other actions globally

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Early May saw the U.S. Food and Drug Administration (FDA) expand the approved use of Bayer’s Stivarga (regorafinib) to include treatment of patients with hepatocellular carcinoma (HCC or liver cancer) who have been previously treated with the drug sorafenib, marking the first FDA-approved treatment for a liver cancer in almost a decade.
“Limited treatment options are available for patients with liver cancer,” said Dr. Richard Pazdur, acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research and director of the FDA’s Oncology Center of Excellence. “This is the first time patients with HCC have had an FDA-approved treatment that can be used if their cancer has stopped responding to initial treatment with sorafenib.”
This Stivarga application was granted Priority Review designation as well as Orphan Drug Designation.

PRO 140 deemed too broad to be an orphan drug
VANCOUVER, Wash.—In mid-April, CytoDyn Inc., a biotechnology company focused on the development of new antibody therapies for combating human immunodeficiency virus (HIV) infection, announced that its application for Orphan Drug Designation (ODD) was not granted by the Office of Orphan Products Development of the FDA because PRO 140 appears to have the potential to treat more than just the subset of multidrug-resistant HIV patients for which the designation was requested.
CytoDyn is currently conducting a pivotal Phase 2b/3 trial with PRO 140 in combination with other antiretroviral agents in the patient population submitted to the FDA in the ODD application. However, CytoDyn is also conducting a 300-patient Phase 2b/3 trial with PRO 140 as a single-agent maintenance therapy for HIV-infected patients, which is a U.S. patient population that far exceeds the 200,000-patient threshold for ODD.

Tecentriq wins FDA approval for advanced bladder cancer
SOUTH SAN FRANCISCO, Calif.—Genentech, a unit of Swiss drugmaker Roche Holding AG, announced recently approval from the FDA for its already approved immunotherapy drug, Tecentriq, to treat advanced bladder cancer.
Tecentriq, also known as atezolizumab, received the go-ahead under the FDA’s accelerated approval program as a first-line treatment for patients with advanced bladder cancer who are not eligible for standard cisplatin chemotherapy. The drug was earlier approved in patients with advanced or metastatic bladder cancer whose disease worsened within a year of receiving chemotherapy.
The approval, which came six months after the FDA approved Tecentriq for the treatment of non-small cell lung cancer, is a boost to the Swiss drugmaker’s bid to expand indications for the drug. Tecentriq belongs to a closely watched class of drugs called PD-1 inhibitors, which help the immune system fight cancer by blocking a mechanism that tumors use to evade attack.

Regulatory pathway opens for LDL-C-lowering indication for bempedoic acid
ANN ARBOR, Mich.—March saw Esperion Therapeutics Inc., a lipid management company focused on developing and commercializing convenient, complementary, cost-effective, once-daily, oral therapies for the treatment of patients with elevated low-density lipoprotein cholesterol (LDL-C), announce that the FDA recently confirmed that Esperion’s LDL-C- lowering program is adequate to support approval of an LDL-C lowering indication for bempedoic acid.
Esperion plans to submit a New Drug Application by the first half of 2019 for an LDL-C-lowering indication based on the successful completion of the global pivotal Phase 3 program. The proposed product label would include specific language for use of bempedoic acid as an adjunct to maximally tolerated statin therapy in patients with hypercholesterolemia, specifically those at high cardiovascular disease risk with atherosclerotic cardiovascular disease and/or heterozygous familial hypercholesterolemia who require additional LDL-C lowering.
“We are very pleased to have achieved clarity from FDA regarding Esperion’s LDL-C-lowering development program,” said Tim M. Mayleben, president and CEO of Esperion. “Our experienced lipid management team has worked closely with regulatory authorities and our key advisors to achieve this encouraging outcome.”

Marketing authorization granted to Darzalex
COPENHAGEN, Denmark—Genmab A/S announced in early May that the European Commission had granted European Marketing Authorization for Darzalex (daratumumab) in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy.
“We are very pleased that Darzalex is now approved in Europe in combination with other standard multiple myeloma therapies and that a far greater number of patients suffering from this incurable disease will now have access to this first-in-class immunotherapy,” said Dr. Jan van de Winkel, CEO of Genmab.
The decision was based on data from two Phase 3 studies: the CASTOR study of daratumumab in combination with bortezomib and dexamethasone vs. bortezomib and dexamethasone alone in patients with relapsed or refractory multiple myeloma (published in The New England Journal of Medicine in August 2016) and the POLLUX study of daratumumab in combination with lenalidomide and dexamethasone vs. lenalidomide and dexamethasone alone in patients with relapsed or refractory multiple myeloma (published in the New England Journal of Medicine in October 2016). Data from two early-stage studies also supported the approval: the Phase 1 MMY1001 study (daratumumab in combination with pomalidomide and dexamethasone) and the Phase 1/2 GEN503 study (daratumumab in combination with lenalidomide and dexamethasone).

FDA clears IND application for UCART19
SERVIER, France—Servier, together with Pfizer Inc. and Cellectis, announced in March that the FDA had granted Servier Investigational New Drug clearance to proceed in the United States with the clinical development of UCART19, an allogeneic, gene-edited cellular therapy candidate to treat relapsed/refractory acute lymphoblastic leukemia.
Servier is sponsoring the CALM Phase 1 study on UCART19. In 2015, Servier acquired exclusive rights from Cellectis for UCART19, which is being co-developed by Servier and Pfizer.

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