States take action to preserve access to health coverage | Follow-on funding pulls in $175.5M for RegenxBio | Amicus' Fabry disease drug gets FDA nod
August 13, 2018
Specialty Pharmacy SmartBrief
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Leading News
States take action to preserve access to health coverage
Tired of waiting for the Trump administration to solve their health care challenges, state governments are taking matters into their own hands to ensure residents have access to health care coverage. Some of the actions taken by states include expanding Medicaid eligibility, establishing mandates that individuals enroll in health coverage, introducing bills to address prescription drug prices and approving measures to restrict access to short-term health plans.
U.S. News & World Report (8/10) 
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Specialty Pharmacy Update
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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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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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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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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Legislation & Regulation
Azar looks to overhaul Medicaid drug-rebate guidelines
HHS Secretary Alex Azar said the department is looking to change Medicaid's drug-rebate guidelines to prevent drugmakers from gaming the program, and it will start by blocking pharmaceutical firms from using line extensions to reduce the rebates they have to pay to states. Speaking at an event of the American Legislative Exchange Council, Azar said he also plans to address certificate-of-need rules, which serve as a "significant barrier to new competition and lower-cost market disruptors."
Modern Healthcare (tiered subscription model) (8/9) 
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Hatch, Walden question proposal to limit Rx drug rebates
Senate Finance Committee Chairman Orrin Hatch, R-Utah, and House Energy and Commerce Committee Chairman Greg Walden, R-Ore., are asking the Office of Management and Budget to conduct a full economic analysis of a proposal that could remove legal protections for rebates on the sales of prescription drugs.
The Hill (8/9) 
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HHS chief urges states not to restrict access to short-term health plans
HHS Secretary Alex Azar urged state regulators not to restrict access to short-term health plans because they are viable alternatives for people who can't afford Affordable Care Act plans. A week after the Trump administration issued a final rule extending the duration of short-term health plans from three months to up to three years, several states including Vermont and Maryland already took action to restrict the plans' sales by signing laws limiting their duration to three months, with no opportunities for renewals or extensions.
The Hill (8/9) 
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Clinical & Research News
OncoPep launches Phase Ib study for smoldering myeloma vaccine
A multi-site Phase Ib clinical trial has been initiated by biotech firm OncoPep to assess the safety and tolerability of its experimental vaccine candidate PVX-410 to treat smoldering multiple myeloma. Two different combinations will be tested in about 20 patients, with the first consisting of PVX-410 plus Celgene's investigational histone deacetylase inhibitor citarinostat and the second a combination of PVX-410, citarinostat and lenalidomide.
Drug Development Technology (UK) (8/10) 
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Hot Topics
Trends & Technology
Alnylam Pharmaceuticals' gene-silencing drug receives FDA approval
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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It's bad policy to speculate on what you'll do if a plan fails, when you're trying to make a plan work.
Condoleezza Rice,
diplomat and political scientist
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