Vertex's Symdeko approved for young patients with cystic fibrosis | FDA OKs drug to boost sexual desire in premenopausal women | FDA approves Ocular sNDA for Dextenza
June 24, 2019
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Vertex's Symdeko approved for young patients with cystic fibrosis
Vertex Pharmaceuticals received FDA approval for its Symdeko tablets, a formulation of tezacaftor and ivacaftor, for treatment of pediatric patients ages 6 and older with cystic fibrosis who have certain genetic profiles. Studies found that the safety profile of the drug in patients as young as 6 mirrors the safety profile seen in older children.
Medscape (free registration) (6/21) 
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Health Care & Policy
FDA OKs drug to boost sexual desire in premenopausal women
The FDA has approved Vyleesi, a treatment developed by Amag Pharmaceuticals and Palatin Technologies to restore sexual desire in premenopausal women. Amag will give Palatin $60 million for the regulatory decision and additional payments for milestones and royalties.
Reuters (6/21) 
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FDA approves Ocular sNDA for Dextenza
The supplemental new drug application for the use of Ocular Therapeutix's Dextenza, or dexamethasone ophthalmic insert, as a treatment of ocular inflammation after opthalmic surgery has been approved by the FDA. The decision was backed by late-stage trials in which more patients experienced no pain eight days after treatment with Dextenza and demonstrated a statistically significant lack of anterior chamber cells versus the vehicle group 14 days after treatment.
eMPR (6/21) 
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Allergan secures FDA nod for Botox in pediatric cerebral palsy
The FDA has approved Allergan's supplemental biologics application for Botox as a treatment for children two to 17 years old with upper limb spasticity.
The Irish Times (Dublin) (6/24) 
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Updated data reported for Dicerna's primary hyperoxaluria candidate
Dicerna Pharmaceuticals unveiled updated results from an ongoing early-stage trial assessing the safety, efficacy and tolerability of its experimental drug DCR-PHXC in patients with type 1 or type 2 primary hyperoxaluria. The data, which showed patients experiencing substantial 24-hour reductions in urinary oxalate levels after administration of a single-dose of DCR-PHXC, were presented during the Oxalosis & Hyperoxaluria Foundation International Hyperoxaluria Workshop.
Seeking Alpha (6/21) 
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Hyperkalemia drug beneficial to dialysis patients with ESRD
Data shows that AstraZeneca's Lokelma, or sodium zirconium cyclosilicate, was effective in treating patients with end-stage renal disease who have hyperkalemia and are undergoing hemodialysis. Findings were presented at the 56th Congress of the European Renal Association-European Dialysis and Transplant Association and were also published in the Journal of the American Society of Nephrology.
Medscape (free registration) (6/21) 
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Company & Financial News
Sites chosen for international trial of CMV vaccine candidate
Hookipa Pharma will fund a clinical trial for its investigational vaccine HB-101 for the prevention of cytomegalovirus infections in patients who undergo kidney transplants. The University of Cincinnati Medical Center will serve as one of an estimated 40 international test sites that will enroll a total of 150 transplant patients.
Precision Vaccinations (6/21) 
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4 life sciences startups launch IPOs
Life sciences startups had a big day of initial public offerings on Wednesday. The genome sequencing and analysis platform Personalis raised $134 million; biotech Akero Therapeutics raised about $92 million; gene therapy developer Prevail Therapeutics raised $125 million; and immune therapy developer Atreca raised $113.8 million.
Xconomy (6/20) 
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Food & Agriculture
Crop Life exhibits benefits of biotech maize
The Crop Life Pakistan Association exhibited the benefits of biotechnology through the performance of maize hybrids in the field and highlighted its importance in cultivation during a value assessment trial.
Online International News Network (Pakistan) (6/22) 
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Industrial & Environmental
Trump seeks revisions to EPA's expanded biofuel waiver program
President Donald Trump has reportedly instructed cabinet members to explore changes to the Environmental Protection Agency's biofuel waiver program for oil refiners in response to complaints from the ethanol industry. The EPA has issued four times the number of small refinery waivers since Trump became president, upsetting corn farmers.
Reuters (6/20) 
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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
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