Clinical Intelligence

CSL Behring receives Orphan Drug exclusivity for Haegarda

Monday, July 24, 2017

CSL Behring announced that the FDA has granted the company seven years of orphan drug exclusivity for Haegarda (C1 Esterase Inhibitor Subcutaneous [Human]), the first and only subcutaneous treatment option for prevention of hereditary angioedema (HAE) attacks. Haegarda was approved by the FDA on June 22, 2017 for routine prophylaxis to prevent HAE attacks in adolescent and adult patients, and marketing exclusivity will continue through June 22, 2024.

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Samumed completes phase I chronic tendinopathy study

Monday, July 24, 2017

Samumed, a leader in musculoskeletal conditions, announced the successful completion of a phase I clinical trial in healthy subjects for its potential treatment of chronic tendinopathy, a degenerative and fibrotic condition caused by injuries or overuse and that has no FDA-approved drug treatments today. The study results supported the continuation of the program into future studies in tendinopathy patients. There were no serious adverse events related to the drug (as deemed by the investigators) and a maximum tolerated dose was not reached in any of the three treatment groups. A detailed analysis, including safety and pK results, will be presented at a future medical conference.

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FDA accepts Amgen’s Aimovig BLA for migraine

Monday, July 24, 2017

Amgen announced that the FDA has accepted for review the Biologics License Application (BLA) for Aimovig (erenumab) for the prevention of migraine in patients experiencing four or more migraine days per month. If approved, Aimovig is expected to be the first-and-only monoclonal antibody targeting the calcitonin gene-related peptide (CGRP) receptor, specifically designed for the prevention of migraine.

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FDA Accepts Otonomy’s Otiprio sNDA filing for acute otitis externa

Wednesday, July 19, 2017

Otonomy, a biopharmaceutical company focused on the development and commercialization of innovative therapeutics for diseases and disorders of the ear, announced that its Supplemental New Drug Application (sNDA) for the approval of OTIPRIO as a treatment of acute otitis externa (AOE) has been accepted for filing by the FDA and been assigned a Prescription Drug User Fee Act (PDUFA) action date of March 2, 2018. The acceptance of the sNDA indicates that the application is sufficiently complete to permit a substantive review by the FDA.

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Sangamo receives FDA Fast Track for SB-318a SB-913

Monday, July 17, 2017

Sangamo Therapeutics announced that the FDA has granted Fast Track designation to SB-318 and SB-913, the company’s clinical stage in vivo genome editing product candidates for the treatment of Mucopolysaccharidosis Type I (MPS I) and MPS II, respectively. The FDA’s Fast Track designation is designed to facilitate the development and expedite the review of drugs and biologics to treat serious conditions and fill an unmet medical need. Once a drug receives Fast Track designation, early and frequent communication with the FDA is encouraged throughout the development and review process. The frequency of communication is designed to ensure that questions and issues are resolved quickly, potentially leading to earlier drug approval and access by patients.

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Helius, USAMRMC enroll last subject enrolled in TBI trial

Monday, July 17, 2017

Helius Medical Technologies, a medical technology company focused on the treatment of neurological symptoms caused by disease or trauma, and the United States Army Medical Research and Materiel Command (USAMRMC) announce that the last subject has been enrolled in the registrational clinical trial to investigate the safety and effectiveness of the Portable Neuromodulation Stimulator (PoNS) device for the rehabilitation of chronic balance deficits caused by mild-to-moderate Traumatic Brain Injury (mTBI). The trial is intended to serve as the basis for Helius to submit applications for marketing clearance in the U.S., Canada and Europe for the PoNS device.

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Vertex gains reimbursement agreement in Italy for cystic fibrosis drug Orkambi

Friday, July 14, 2017

Vertex Pharmaceuticals announced that the Italian Medicines Agency (Agenzia Italiana del Farmaco, or AIFA) has agreed to reimburse Orkambi (lumacaftor/ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The agreement is published online in the Italian Official Gazette.

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