Industry News

Merck, known as MSD outside the United States and Canada, announced that the U.S. Food and Drug Administration (FDA) has accepted for review a new supplemental Biologics License Application (sBLA) for KEYTRUDA® (pembrolizumab), the company’s anti-PD-1 therapy, to include data from KEYNOTE-010.

Baxalta Incorporated announced that it has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for approval of ADYNOVI, an extended circulating half-life recombinant Factor VIII (rFVIII) treatment, for pediatric, adolescent and adult patients with hemophilia A and for use during surgery. Currently licensed in the U.S. as ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated] and under regulatory review in Japan, Canada and Switzerland, ADYNOVI is the only rFVIII treatment for hemophilia A developed based on the demonstrated efficacy of ADVATE [Antihemophilic Factor (Recombinant)].

BioMarin Pharmaceutical Inc. announced positive 48-week results from its Phase 1/2 pivotal study for cerliponase alfa, a recombinant human tripeptidyl peptidase 1 (rhTPP1) to treat children with CLN2 disease, a form of Batten disease.  CLN2 disease is a rapidly progressing, fatal neurodegenerative disease with no approved treatments, where the majority of affected children lose their ability to walk and talk by approximately six years of age.

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Sangamo BioSciences, Inc. announced the presentation of new preclinical data in disease models from its In Vivo Protein Replacement Platform™ (IVPRP) programs for MPS I (Hurler syndrome) and MPS II (Hunter syndrome). The data demonstrate that the Company's IVPRP approach enabled stable production of therapeutic levels of replacement enzyme from the liver into the circulation and secondary tissues, including the brain, resulting in significant reduction in biomarkers of the disease and, importantly, statistically significant improvements in cognitive function in treated animals.

Sanofi and its specialty care global business unit Sanofi Genzyme presented data from NEO1, its Phase 1/2 clinical study evaluating the investigational novel enzyme replacement therapy neoGAA in 24 patients with late-onset Pompe disease.

AstraZeneca announced that it has entered into an agreement with ProStrakan Group, a subsidiary of Kyowa Hakko Kirin Co. Ltd., for the rights to Moventig (naloxegol) in the European Union (EU), Iceland, Norway, Switzerland and Liechtenstein. Moventig is the first once-daily, oral peripherally-acting mu-opioid receptor antagonist (PAMORA) approved in Europe for the treatment of opioid-induced constipation (OIC) in adult patients who have had an inadequate response to laxative(s).

BioMarin Pharmaceutical Inc. Announced that BMN 270, an investigational gene therapy for the treatment of patients with hemophilia A, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). BioMarin is currently conducting a Phase 1/2 study to evaluate the safety and efficacy of BMN 270 gene therapy in up to 12 patients with severe hemophilia A and will provide a program update in April.  BMN 270 is an AAV 5 factor VIII vector, designed to restore factor VIII plasma concentrations, essential for blood clotting in patients with hemophilia A.

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Edwards Lifesciences Corporation announced U.S. Food and Drug Administration approval to expand use of the Edwards SAPIEN XT transcatheter heart valve for pulmonic valve replacement procedures.

Roche announced that the US Food and Drug Administration (FDA) approved Gazyva® (obinutuzumab) plus bendamustine chemotherapy followed by Gazyva alone as a new treatment for people with follicular lymphoma who did not respond to a Rituxan® (rituximab)-containing regimen, or had their follicular lymphoma return after such treatment. Follicular lymphoma is the most common type of indolent (slow-growing) non-Hodgkin lymphoma (NHL) and accounts for approximately one in five cases of NHL.

Abeona Therapeutics, Inc. announced the FDA cleared the Company's Investigational New Drug Application for ABO-102 (AAV- SGSH), a single treatment strategy for Mucopolysaccharidosis Type IIIA (MPS IIIA). The ABO-102 IND application is now active and enables Nationwide Children's Hospital (Columbus, OH) to initiate a Phase 1/2 clinical study designed to assess the safety, tolerability and potential efficacy of ABO-102 in children with MPS IIII A.