FDA

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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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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Eureka to start phase I ET190L1-ARTEMIS T cell therapy trial

Monday, November 27, 2017

Eureka Therapeutics, a clinical stage biotechnology company focused on improving the safety profile of T cell therapies and developing novel T cell therapies for the treatment of solid tumors, announced FDA allowance of its investigational new drug (IND) application and authorization to commence a phase I clinical trial for ET190L1-ARTEMIS T cells in Relapsed and Refractory CD19+ Non-Hodgkin Lymphoma (NHL), including Chronic Lymphocytic Leukemia (CLL). Eureka expects to enroll the first patient in this trial in the first quarter of 2018.

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New patient adherence tech tests the limits

Monday, November 27, 2017

With the FDA’s recent approval of Abilify MyCite, a “digital pill” that can indicate whether a patient has taken his or her medication, physicians may finally have an effective way of attacking the problem of medication nonadherence. Abilify MyCite is a drug-device combina-tion product that consists of Otsuka Pharmaceutical’s oral Abilify (aripiprazole) tablets that have been embedded with an ingestible biosensor developed by Proteus Digital Health.

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Fasenra (Benralizumab) receives FDA approval for severe eosinophilic asthma

Friday, November 17, 2017

AstraZeneca and its global biologics research and development arm, MedImmune, announced that the FDA has approved FASENRA (benralizumab) for the add-on maintenance treatment of patients with severe asthma aged 12 years and older, and with an eosinophilic phenotype. FASENRA is not approved for the treatment of other eosinophilic conditions or relief of acute bronchospasm or status asthmaticus.

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