Bio Roundup: Rare Disease Showdowns, Prices on TV, Dealmania & More

Xconomy National — 

The market for drugs for rare or “orphan” diseases continues to grow. An April report from Evaluate Pharma predicted sales of orphan drugs to rise 12 percent a year between now and 2024, when they will account for an estimated 20 percent of worldwide prescription drug sales. The field has gotten so competitive that multiple products are on the market for some diseases.

A few years ago, for instance, there were no drugs available for spinal muscular atrophy (SMA), an inherited and potentially lethal neuromuscular disease. Soon there could be three. Patients with the rare, debilitating transthyretin amyloidosis had no marketed treatments before 2018. This week, the third one was approved in less than a year. As Xconomy has detailed, these instances will leave payers, patients, and clinicians with tough decisions to make.

Elsewhere, drug prices are coming to commercials near you, a new gene therapy is on the precipice of US approval, and a bunch of pharma companies whipped out their checkbooks. Let’s get to it.


—The American Academy of Neurology meeting this week featured multiple presentations on two emerging therapies for SMA: the Novartis gene therapy Zolgensma and the Roche oral liquid risdiplam. Xconomy detailed the results, put them in context, and rounded up some of the other headlines from the meeting.

—The Wall Street Journal delved into healthcare system problems that Zolgensma could trigger if, as expected this month, it becomes just the second approved gene therapy in the U.S.. Analysts have projected a price tag of $2 million for a one-time dose.

—The FDA approved two oral formulations of the Pfizer (NYSE: PFE) drug tafamidis (Vyndaqel, Vyndamax) for heart problems suffered by patients with the rare genetic disease transthyretin amyloidosis. The approval, two months ahead of schedule, sets up a battle between Pfizer, Alnylam Pharmaceuticals (NASDAQ: ALNY), and Akcea Therapeutics (NASDAQ: AKCA).

—AstraZeneca (NYSE: AZN) spinout Viela Bio will seek FDA approval of experimental drug inebilizumab, which is aimed at the rare autoimmune disease neuromyelitis optica spectrum disorder. Viela revealed successful Phase 3 results at AAN. Viela is racing rival therapies from Alexion Pharmaceuticals (NASDAQ: ALXN) and Roche. Here’s more from Vantage.


—The Trump administration ruled that drug companies must include monthly wholesale prices in their television ads. The rule takes effect in 60 days. HHS secretary Alex Azar, previously an executive at Eli Lilly (NYSE: LLY) told reporters that drug companies shouldn’t complain: “If you are ashamed of your drug prices, change your drug prices, it’s that simple.” Politico has more.

—Responding on Twitter to a Texas pediatrician, a Texas state legislator has drawn national attention for calling vaccines “sorcery.”

—The World Health Organization ramped up its Ebola vaccination efforts in the Democratic Republic of the Congo, where containment of an outbreak is being hampered by violence in the region. In addition to a Merck (NYSE: MRK) vaccine, WHO will use an experimental vaccine from Johnson & Johnson (NYSE: JNJ). Science has more.

—The Senate grilled drug makers and pharmacy benefit managers earlier this year about high drug prices, and the chamber has begun producing different versions of pricing reform legislation. The House is having its turn, too, with a hearing this week on the drug supply chain.


—Novartis paid Takeda $3.4 billion for rights to lifitegrast (Xiidra), a treatment for dry eye, which Takeda acquired through its buyout of Shire. The FDA approved lifitegrast in 2016; the drug generated $400 million last year.

—Bristol-Myers Squibb (NYSE: BMY) priced $19 billion in notes to help fund its big buyout of Celgene (NASDAQ: CELG), which is slated to close later this year.

—Sarepta Therapeutics (NASDAQ: SRPT) licensed from Nationwide Children’s Hospital a gene therapy for the most common form of Limb-Girdle muscular dystrophy. It is the ninth gene therapy license or option agreement between … Next Page »

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