United States

FDA announces approval of first breakthrough-designated test

Friday, December 8, 2017

The FDA approved the FoundationOne CDx (F1CDx), the first breakthrough-designated, next generation sequencing (NGS)-based in vitro diagnostic (IVD) test that can detect genetic mutations in 324 genes and two genomic signatures in any solid tumor type. The Centers for Medicare & Medicaid Services (CMS) at the same time proposed coverage of the F1CDx. The test is the second IVD to be approved and covered after overlapping review by the FDA and CMS under the Parallel Review Program, which facilitates earlier access to innovative medical technologies for Medicare beneficiaries.

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FDA approves first biosimilar for certain breast and stomach cancers

Friday, December 8, 2017

The FDA approved Ogivri (trastuzumab-dkst) as a biosimilar to Herceptin (trastuzumab) for the treatment of patients with breast or metastatic stomach cancer (gastric or gastroesophageal junction adenocarcinoma) whose tumors overexpress the HER2 gene (HER2+). Ogivri is the first biosimilar approved in the U.S. for the treatment of breast cancer or stomach cancer and the second biosimilar approved in the U.S. for the treatment of cancer.

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Ra touts positive interim results from paroxysmal nocturnal hemoglobinuria study

Tuesday, December 5, 2017

Ra Pharmaceuticals announced positive interim results from the company’s ongoing, global phase II clinical program evaluating RA101495 SC for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Ra Pharma is a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for the treatment of complement-mediated diseases and is developing RA101495 as a novel, subcutaneously-administered (SC) inhibitor of complement component 5 (C5).

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NIH’s All of Us Research Program, National Library of Medicine partner

Monday, December 4, 2017

NIH’s All of Us Research Program and the National Library of Medicine (NLM) have teamed up to raise awareness about the program, a landmark effort to advance precision medicine. Through this collaboration, the National Network of Libraries of Medicine has received a $4.5 million award to support community engagement efforts by public libraries across the United States and to improve participant access.

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BioMarin sells second Priority Review Voucher for $125M

Friday, December 1, 2017

BioMarin Pharmaceutical announced that it has entered into a definitive agreement to sell the Rare Pediatric Disease Priority Review Voucher (PRV) it obtained in April of this year for a lump sum payment of $125,000,000. The Company received the voucher under a FDA program intended to encourage the development of treatments for rare pediatric diseases. BioMarin was awarded the voucher when it received approval of Brineura, a new biological product for patients with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency, a form of Batten disease. The transaction remains subject to customary closing conditions, including anti-trust review.  

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FDA grants orphan designation for Hemophilic Arthropathy treatment TRM-201

Monday, November 27, 2017

Tremeau Pharmaceuticals, a new pharmaceutical company focused on providing non-opioid pain treatments for rare diseases, announced that the FDA has granted an orphan drug designation, on a first cycle review, for TRM-201 (rofecoxib), a COX-2 selective non-steroidal anti-inflammatory drug (NSAID), for the treatment of degenerative joint disease in patients with hemophilia, also known as hemophilic arthropathy (HA). 

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ACRO urges modernization of the R&D Tax Credit

Wednesday, November 22, 2017

As the Senate begins debate on a tax reform bill, the clinical research industry hopes that attention will be given to a small but substantive issue—updating a tax code provision that has had strong bipartisan support for more than 30 years.

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