FDA Approves ORKAMBI® (lumacaftor/ivacaftor) as First Medicine to Treat the Underlying Cause of Cystic Fibrosis for Children Ages 2-5 Years with Most Common Form of the Disease
August 7, 2018
Vertex Pharmaceuticals Incorporated has announced the U.S. Food and Drug Administration (FDA) has approved ORKAMBI® (lumacaftor/ivacaftor) to include use in children ages 2 through 5 years with cystic fibrosis (CF) who have two copies of the F508del-CFTR mutation, making it the first medicine approved to treat the underlying cause of CF in this population.
August 8, 2018
Paratek Pharmaceuticals, Inc. has announced that the Antimicrobials Drug Advisory Committee of the U.S. Food and Drug Administration voted in favor of the approval of intravenous (IV) and oral omadacycline for the treatment of acute bacterial skin and skin structure infections (ABSSSI) (17-1) and community-acquired bacterial pneumonia (CABP) (14-4). Omadacycline is a modernized tetracycline being developed as a once-daily IV and oral, broad spectrum antibiotic for the treatment of serious community-acquired infections.
Ambys Medicines and Takeda Announce Partnership to Pioneer First-In-Class Therapies for the Treatment of Serious Liver Diseases
August 8, 2018
Ambys Medicines and Takeda have announced that they have entered into a partnership to support the advancement of the Ambys platform and pipeline. Ambys is pioneering the application of novel modalities, including cell and gene therapy and gain-of-function drug therapy, to meet the urgent need for treatments that restore liver function and prevent the progression to liver failure across multiple liver diseases that are untreatable or poorly treated today.
August 10, 2018
Gemphire Therapeutics Inc., a clinical-stage biopharmaceutical company focused on developing and commercializing therapies for cardiometabolic disorders, including dyslipidemia and nonalcoholic steatohepatitis (NASH), has announced that the Data and Safety Monitoring Board (DSMB) at Emory University School of Medicine overseeing the investigator-led open label Phase 2a proof-of-concept trial evaluating gemcabene in pediatric patients with non-alcoholic fatty liver disease (NAFLD) has recommended that the trial be terminated due to unanticipated problems.
Astellas Announces Acquisition of Quethera – Acquisition furthers Astellas’ commitment to innovation in ophthalmology with addition of novel gene therapy program for glaucoma
Aug 10, 2018
Astellas Pharma Inc. and Quethera Limited have announced that Astellas has acquired Quethera, a gene therapy company headquartered in the United Kingdom, that is focused on developing novel treatments for ocular disorders, such as glaucoma.
Alnylam Announces First-Ever FDA Approval of an RNAi Therapeutic, ONPATTRO™ (patisiran) for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults
August 10, 2018
Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, has announced that the United States Food and Drug Administration (FDA) approved ONPATTRO™ (patisiran) lipid complex injection, a first-of-its-kind RNA interference (RNAi) therapeutic, for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. ONPATTRO is the first and only FDA-approved treatment for this indication. hATTR amyloidosis is a rare, inherited, rapidly progressive and life-threatening disease with a constellation of manifestations. In addition to polyneuropathy, hATTR amyloidosis can lead to other significant disabilities including decreased ambulation with the loss of the ability to walk unaided, a reduced quality of life, and a decline in cardiac functioning. In the largest controlled study of hATTR amyloidosis, ONPATTRO was shown to improve polyneuropathy – with reversal of neuropathy impairment in a majority of patients – and to improve a composite quality of life measure, reduce autonomic symptoms, and improve activities of daily living.
Regeneron Provides Regulatory Update on EYLEA® (aflibercept) Injection sBLA in Wet Age-Related Macular Degeneration
August 13, 2018
Regeneron Pharmaceuticals, Inc. has announced that due to ongoing labeling discussions the U.S. Food and Drug Administration (FDA) has issued a complete response letter (CRL) regarding the supplemental Biologics License Application (sBLA) for EYLEA® (aflibercept) Injection in patients with wet age-related macular degeneration (wet AMD). The sBLA was based on the second-year data from the VIEW studies during which patients were treated with a modified 12 week dosing schedule (doses given at least every 12 weeks and additional doses as needed). Regeneron expects to complete these discussions and receive a final FDA action within approximately two months.
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