United States

Report: U.S. specialty pharma’s M&A focus shifts to pipelines and innovative products

Tuesday, June 14, 2016

Despite the ongoing challenges weighing on the U.S. specialty pharmaceutical sector, companies will steadily resume M&A with a more refined focus, said Moody’s Investors Service. The ratings agency said it expects acquisitive specialty companies will increase their focus on products with unmet medical need, companies with pipeline drugs and those providing geographic expansion.

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Seattle Children’s leukemia trial shows 93% complete remission rate

Friday, June 10, 2016

Seattle Children’s has announced that 39 of 42 patients treated in a phase I clinical trial using genetically reprogrammed T cells to treat relapsed or refractory acute lymphoblastic leukemia (ALL) have achieved complete remission, showing no detectable leukemia cells in the most sensitive tests. Patients treated in the trial, known as Pediatric Leukemia Adoptive Therapy-02 (PLAT-02), had less than a 20% chance of survival upon enrollment using current treatments. Seattle Children’s will launch phase II of the trial this month, with the aim to enroll 70 patients in the next year.

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Rho named Best CRO by Triangle Business Journal

Monday, June 6, 2016

Rho, a full-service CRO focused on bringing new products to market through a full range of product development services, recently announced that the company was named the 2016 Best CRO by Triangle Business Journal, a multimedia source for local business news, research and events in the Raleigh, Durham and Chapel Hill region of North Carolina, where Rho is headquartered. Rho was also honored with this award in 2015.

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FDA grants Accelerated Approval to Ocaliva for primary biliary cirrhosis

Wednesday, June 1, 2016

Intercept Pharmaceuticals, a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat non-viral, progressive liver diseases, has announced that the FDA has granted accelerated approval to Ocaliva (obeticholic acid) for the treatment of primary biliary cholangitis, previously known as primary biliary cirrhosis (PBC), in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA. Ocaliva is an agonist of the farnesoid X receptor (FXR), a nuclear receptor expressed in the liver and intestine and a key regulator of bile acid, inflammatory, fibrotic and metabolic pathways. 

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Flatiron Health, FDA collaborate on cancer research

Wednesday, June 1, 2016

Flatiron Health and the FDA have signed a research collaboration agreement to determine how real-world evidence derived from de-identified, HIPAA-compliant patient data captured outside of clinical trials can provide new insights into the safety and effectiveness of emerging anti-cancer therapies such as immunotherapeutic agents.

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Senate approves brain cancer research Bill

Tuesday, May 31, 2016

The Senate Committee on Appropriations has approved legislation that provides for the inclusion of “brain cancer” among the disorders eligible for study by the Department of Defense‘s (DoD) Peer Reviewed Cancer Research Program (PRCRP). This provision was included in the fiscal year 2017 Defense Appropriations Act, and mirrors action taken by the House on May 11 to add brain cancer to the PRCRP in its version of the Defense Appropriations Act.

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FDA approves CSL Behring’s AFSTYLA for hemophilia A

Tuesday, May 31, 2016

CSL Behring has announced that the FDA has approved AFSTYLA [Antihemophilic Factor (Recombinant), Single Chain], its novel long-lasting recombinant factor VIII single-chain therapy for adults and children with hemophilia A. AFSTYLA is the first and only single-chain product for hemophilia A that is specifically designed for long-lasting protection from bleeds with two to three times weekly dosing.

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FDA approves first buprenorphine implant for opioid dependence

Friday, May 27, 2016

The FDA has approved Probuphine, the first buprenorphine implant for the maintenance treatment of opioid dependence. Probuphine is designed to provide a constant, low-level dose of buprenorphine for six months in patients who are already stable on low-to-moderate doses of other forms of buprenorphine, as part of a complete treatment program.

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