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U.S. regulators have approved the most expensive medicine ever, for a rare disorder that destroys a baby's muscle control and kills nearly all of those with the most common type of the disease within a couple of years. The treatment is priced at $2.125 million. Out-of-pocket costs for patients will vary based on insurance coverage. (Johnson, 5/24)

The therapy, to be sold as Zolgensma, alters the underlying genetic cause of spinal muscular atrophy and may permanently stop the disease. It is among the first of a host of gene therapies that promise a cure for deadly inherited conditions. “We feel we’re on a path where we hope one day to be able to bring S.M.A. almost to elimination,” said Vas Narasimhan, the Novartis chief executive, in a conference call with reporters. Many drugs for rare diseases have arrived on the market recently costing hundreds of thousands of dollars a year. But few have crossed the $1 million threshold. (Thomas, 5/24)

Critics noted that the bulk of Zolgensma's development was done by AveXis, which Novartis bought last year. “The question in drug pricing isn’t how much is a life worth; it’s what makes a fair return on an investment in R&D and an accessible price,” said David Mitchell, president of Patients for Affordable Drugs, an advocacy group. (Owermohle, 5/24)

Gene therapies promise the chance to cure diseases whose diagnoses were death sentences, but the prices for the first few to be greenlighted raise concerns about whether they can be afforded by governments and health insurers that have been struggling to control health spending. (Roland, 5/24)

Despite high initial costs, gene therapies are expected to save health-care systems money by eliminating the need for lifelong treatment. While manufacturers propose spacing out bills over time, governments and insurers are still trying to figure out how to pay for the treatments and wrestling with uncertainty over their benefits and safety in the long run. (Paton and Cortez, 5/24)

Novartis is likely to face backlash from critics who believe charging millions of dollars for any medicine — no matter how effective — renders it unaffordable for a healthcare system already under financial stress. There’s also competition. Spinraza, approved in late 2016 and sold by Biogen, has already been used to successfully treat thousands of patients with severe and milder forms of SMA. The drug requires regular spinal infusions costing $750,000 in the first year and $375,000 annually thereafter, for life. Sales last year totaled $1.7 billion. Zolgensma may be more convenient than Spinraza, but Roche is developing a daily pill for SMA called risdiplam that could reach the market in 2020. (Feuerstein, 5/24)

[F]or those patients lucky enough to get it, it appears it can save their lives with a one-time treatment.Three-year-old Donovan Weisgarber is one of those patients. When he was born he seemed perfectly healthy. But within weeks, it became clear something was terribly wrong. "It was about when he was about one month old that when we started to notice some symptoms," says his mother, Laura Weisgarber, 32, of Columbus, Ohio. (Stein, 5/24)