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Bio Roundup: Big Approvals, ICER Influence, Drug-Price Pushback & More

Xconomy National — 

[Updated 8/17/18, 10:21 a.m. See below.] We’ll start the roundup this week with two drug approvals that came late last Friday. Both were landmarks for the companies receiving the nod, Alnylam Pharmaceuticals and Amicus Therapeutics.

We also saw a new biotech emerge in the muggy New York heat, a couple deals for new flu vaccines, and interesting developments in the complicated world of drug pricing.

It’s hot out there. If you missed some headlines this week, don’t sweat it. Stay cool with the roundup.

FDA SAYS YES… AND NO

—The FDA approved the first-ever RNAi medicine, a drug from Alnylam Pharmaceuticals (NASDAQ: ALNY), for the rare disease hereditary transthyretin amyloidosis. Alnylam priced it at $450,000 per year but said the actual price would be tied to its performance, starting with one deal it reached in principle with Harvard Pilgrim Healthcare. Xconomy profiled what the FDA approval would mean for patients, clinicians, and payers earlier this month.

—Amicus Therapeutics (NASDASQ: FOLD) finally won U.S. approval for its Fabry disease drug migalastat (Galafold) after years of regulatory ups and downs with the FDA. Amicus priced the drug at $315,000 a year and promised not to boost its price more than consumer inflation. CEO John Crowley spoke with Xconomy about the company’s rollercoaster ride to approval.

—The FDA approved a generic version of the EpiPen, an auto-injector that administers epinephrine to treat allergic reactions, developed by Teva Pharmaceutical (NYSE: TEVA).

—Regeneron Pharmaceuticals (NASDAQ: REGN) said on Monday that due to “ongoing label discussions,” the FDA rejected a version of its age-related macular degeneration drug aflibercept (Eylea) that requires less frequent dosing. But in a quick turnaround, the Tarrytown, NY, drug maker said Friday that the regulator approved its application for the use of the drug on a 12-week dosing schedule. [Item updated with approval decision issued Friday morning.]

MORE REGULATION AND DATA

—Fasinumab, a pain drug that Regeneron Pharmaceuticals and partner Teva hope will offer an alternative to addictive opioids, posted encouraging early results in a Phase 3 study. But fasinumab’s safety will be under tight scrutiny for the remainder of the study because of risks seen in earlier testing.

—GlaxoSmithKline’s Viiv Healthcare division said its injectable two-drug combination to treat HIV infection passed a Phase 3 study but did not release details.

—The FDA released guidance to speed up human trials for some new cancer drugs. The goal is to compress the traditional three phases of clinical studies into one continuous study, but only for “patients with serious diseases for which no curative therapies are available,” the FDA document says.

—The agency and the National Institutes of Health are also proposing new measures to streamline the review of gene therapies. Here’s more from Regulatory Affairs Professional Society.

—Some of the world’s biggest tech companies pledged to work together on healthcare data standards.

THE PRICE YOU PAY

—Starting next year, CVS Health, which owns one of the biggest negotiators of drug prices in the U.S., will use nonprofit drug-price watchdog ICER’s evaluations to help its clients—insurers and employers—decide which drugs to cover. ICER’s formulas have drawn ire from the biopharma industry. This is the first time ICER’s data will determine coverage.

—CVS also said on an earnings call that it keeps 2 percent of the rebates that it negotiates with drug companies and passes the rest back to its clients. That figure is lower than what CVS told the Trump administration earlier this summer, as Fierce Healthcare noted. Drug companies have taken plenty of heat, but so-called PBMs like CVS Caremark and Express Scripts are also under scrutiny at the federal and state levels for their role in high drug prices.

— Express Scripts (NASDAQ: ESRX), meanwhile, is working with biotechs like BioMarin Pharmaceutical (NASDAQ: BMRN) and Bluebird Bio (NASDAQ: BLUE) on “exclusive arrangements” to distribute their gene therapies through its specialty pharma business, Reuters reported.

—The National Institute for Health and Care Excellence, the U.K.’s drug pricing watchdog, said the spinal muscular atrophy drug nusinersen (Spinraza) is not cost-effective, despite a discount offered by the drug’s owner Biogen (NASDAQ: BIIB). Here’s more from Reuters.

—Kaiser Health News weighed in on the Trump administration’s efforts to rein in drug prices.

SUMMERTIME DEALS

—Four months after acquiring GlaxoSmithKline’s gene therapy portfolio, Orchard Therapeutics closed a $150 million Series C round backed by a group of “crossover” investors, typically a signal that a private company is preparing to go public.

—Pfizer (NYSE: PFE) will pay BioNTech of Germany $120 million upfront to develop flu vaccines using messenger RNA technology. Sanofi and Translate Bio (NASDAQ: TBIO) cut a smaller deal for similar products earlier this summer.

—Biotech creator Accelerator launched its latest New York startup, Magnolia Therapeutics, with a $31 million round and plans to develop drugs for neurodegenerative diseases. The startup’s research comes from the University of Texas MD Anderson Cancer Center.

—Novo Nordisk (NYSE: NVOacquired an experimental diabetes treatment from University of Bristol spinout Ziylo. Novo didn’t say how much it paid up front, just that the deal could be worth more than $800 million if various milestones and sales targets are hit.

NAMES AND FACES

—The Broad Institute’s Sekar Kathiresan and colleagues said they have identified a swath of genetic variations that indicate higher risk of cardiovascular disease and should be put to use in a genetic test. Their work was published in Nature Genetics.

—Former Voyager Therapeutics CEO and one-time Eli Lilly R&D chief Steven Paul was named CEO of Karuna Pharmaceuticals.

—Gilead Sciences chief medical officer Andrew Cheng became the latest longtime Gilead executive to announce a departure.

—Brad Loncar, who created the first stock index to track cancer immunotherapy, has launched a new index based on Chinese biotech companies.

Ben Fidler and Frank Vinluan contributed to this report. 

Photo courtesy of Ano Lobb via Creative Commons 2.0.