FDA OKs first gene-silencing drug for polyneuropathy of hATTR amyloidosis | Amicus' Fabry disease drug gets FDA nod | Oximetry shows promise in identifying adenotonsillectomy need in pediatric SDB
August 13, 2018
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FDA OKs first gene-silencing drug for polyneuropathy of hATTR amyloidosis
The FDA has approved Alnylam Pharmaceuticals' Onpattro, or patisiran, for the treatment of polyneuropathy in patients with hereditary ATTR amyloidosis, making it the first gene-silencing drug to win US approval. Onpattro will carry an annual list price of $450,000 for the average patient, and several insurers, including Harvard Pilgrim Health Care, agreed to cover the drug through value-based deals with Alnylam.
Reuters (8/10) 
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Health Care & Policy
Amicus' Fabry disease drug gets FDA nod
Amicus Therapeutics' Galafold, or migalastat, was approved by the FDA as a treatment for adult patients with Fabry disease. The company will market the drug immediately.
Seeking Alpha (8/10) 
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Oximetry shows promise in identifying adenotonsillectomy need in pediatric SDB
Youths with sleep-disordered breathing who underwent adenotonsillectomy had a significantly greater median decline in the number of oxygen desaturation episodes per hour of at least 3% by nocturnal oximetry from baseline to three months' follow-up, and were significantly more likely to achieve normal ODI3 at follow-up, compared with those in the control group, Greek researchers reported in Pediatrics. The findings also found the adenotonsillectomy group had significantly greater improvements in disease-specific quality-of-life scores at three months than the control group.
Medscape (free registration)/Reuters (8/10) 
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Scientists study antibodies' potential therapeutic value against Ebola
An international group of researchers from the Viral Hemorrhagic Fever Immunotherapeutic Consortium is working to determine which Ebola-fighting antibodies are best and why to create a combined therapeutic cocktail that could disable the infection machinery of the Ebola virus and trigger the patient's immune system to combat the virus. The research was published in Cell.
Genetic Engineering & Biotechnology News (8/10) 
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FDA OKs Cipla's generic Reyataz
The FDA approved Cipla's abbreviated new drug application for atazanavir capsules in 100 mg, 150 mg, 200 mg and 300 mg, the AB-rated generic therapeutic equivalent version of Bristol-Myers Squibb's Reyataz. The drug, in combination with antiretroviral agents, is indicated to treat patients age 6 and older with HIV-1 infection who weigh at least 15 kg.
PharmaBiz (India) (8/13) 
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BeiGene launches late-stage trial for NSCLC drug
The first patient was dosed in a late-stage trial of BeiGene's tislelizumab in combination with chemotherapy, being assessed in China as a treatment of stage IIIB or stage IV squamous non-small cell lung cancer. The study will evaluate progression-free survival as assessed by the Independent Review Committee.
PharmaBiz (India) (8/13) 
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Company & Financial News
Ophthalmic gene therapy firm Quethera purchased by Astellas
Ophthalmic gene therapy company Quethera was purchased by Astellas Pharma in a deal that gives Quethera upfront and contingent payments of up to $110.3 million. Quethera's lead program is an undisclosed gene therapy in preclinical testing for patients with glaucoma.
BioCentury (8/10) 
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Follow-on funding pulls in $175.5M for RegenxBio
A follow-on funding pulled in $175.5 million for RegenxBio, which has an ongoing Phase I/II study of RGX-501 for the treatment of patients with homozygous familial hypercholesterolemia.
BioCentury (8/10) 
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Food & Agriculture
Is gene editing the future of agriculture?
Startups such as Calyxt are leading the way as companies apply gene-editing techniques to soybeans and other crops. Proponents say these techniques allow for more-precise DNA modification, but it remains to be seen how regulators and the public will react.
Reuters (8/10) 
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Industrial & Environmental
Researchers use CRISPR to improve biofuel yield using yeast
By using the CRISPR gene-editing tool to alter two of the more than 12 million nucleotides in the yeast genome, scientists from the University of Wisconsin-Madison and the Department of Energy were able to protect yeast from damage caused by pretreatment chemicals in biofuel production. The ionic liquids used in biofuel production can render yeast up to 70 percent less effective at converting sugar to biofuel.
Biofuels Digest (8/11) 
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The news summaries appearing in BIO SmartBrief are based on original information from news organizations and are produced by SmartBrief, Inc., an independent e-mail newsletter publisher. The information is not compiled or summarized by BIO. Questions and comments should be directed to SmartBrief at bio@smartbrief.com.
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