FDA approves first treatment targeting muscle loss in spinal muscular atrophy

Scholar Rock’s Isembyld (apitegromab-mstn) has received FDA approval as a treatment for spinal muscular atrophy, a rare, severe neuromuscular disease that leads to irreversible loss of motor neurons and progressive muscle wasting.

The Cambridge, Mass.-based biopharma company said Isembyld is the first and only muscle-targeted treatment to demonstrate motor function improvement in individuals with spinal muscular atrophy who are receiving treatment with a drug that targets the survival motor neuron 2 (SMN2) back-up gene, such as Biogen’s Spinraza (nusinersen) or Genentech’s Evrysdi (risdiplam).

Isembyld, a myostatin inhibitor, is indicated for patients ages 2 years and older who are receiving SMN2-targeted treatment.

“Today’s FDA approval of Isembyld marks a defining moment for the [spinal muscular atrophy] community, as we now launch the world’s first-ever muscle-targeted treatment for children and adults living with [spinal muscular atrophy] in the U.S.,” Scholar Rock’s CEO, David Hallal, said in a Sept. 11 press release.

“After decades of failed industry-wide efforts to unlock the potential of myostatin inhibition, Scholar Rock has delivered a therapeutic breakthrough with Isembyld,” Hallal said.

Kenneth Hobby, president of Cure SMA, said, “The approval of Isembyld as the first-ever treatment to directly target the muscular component of [spinal muscular atrophy] is a significant turning point for adults and children who have been waiting for innovative therapeutic options to improve motor function.”

“Such improvements are fundamental to maintaining independence and to enabling participation in important activities of daily living, from self-care to work and social interactions,” Hobby added.

Scholar Rock noted in the press release that Isembyld would be “available to ship in the coming days.”

The drug is administered every four weeks as an intravenous infusion. Dosing is determined by the patient’s age and weight.

According to MedCity News, Scholar Rock has priced Isembyld at $11,659 per vial and estimates an annual net price of approximately $310,000 after taking into account patient compliance as well as payer rebates and discounts.

Isembyld is Scholar Rock’s first drug to receive FDA approval.

Spinal muscular atrophy affects approximately 1 in 10,000 live births and is among the leading genetic causes of infant mortality, according to the FDA. The disease is caused by mutations in the SMN1 gene that leaves individuals with insufficient quantities of a protein essential for motor neuron survival.

A back-up gene, SMN2, typically produces a faulty version of the protein. Drugs such as Spinraza and Evrysdi correct the defect in the SMN2 gene, allowing the body to produce enough of the protein to keep motor neurons alive, the FDA explained.

“While these treatments have significantly improved outcomes for many patients, those with more advanced disease continue to experience substantial motor limitations (including the ability to walk or move independently), highlighting the need for therapies that directly address muscle loss,” the agency stated in its announcement of Isembyld’s approval.

Novartis’ Zolgensma (onasemnogene abeparvovec-xioi) gene therapy delivers a functioning version of the SMN1 gene, but the treatment can only be administered to patients under the age of 2 years.

As muscle weakness progresses in patients with spinal muscular atrophy, their ability to walk, move, and breathe is affected. Eventually, the loss of muscle function becomes fatal.

MedCity News reported that additional clinical research of apitegromab is underway that could expand the drug’s label — possibly for use in patients younger than 2, for instance.

Apitegromab has also been evaluated in a midstage trial in combination with Eli Lilly’s Zepbound (tirzepatide) to determine whether it might help prevent the loss of muscle mass, a side effect commonly seen with GLP-1 drugs. The study results showed the combination significantly preserved lean muscle mass relative to treatment with tirzepatide and placebo.

Results from the Phase III SAPPHIRE study, which supported the approval of Isembyld, were published a year ago in The Lancet Neurology.

Along with the approval of Isembyld, Scholar Rock was awarded a priority review voucher, which the company could use to accelerate the review of another rare disease treatment. MedCity News reported, however, that the company’s CFO said Scholar Rock plans to sell the voucher instead. Similar vouchers have recently been sold for close to $200 million.

HCR #220: A conversation with Dr. Marc Harrison, Chair, TowerBrook Healthcare Institute

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What else you need to know

Novo Nordisk is rebranding, dropping “Nordisk” from its name for day-to-day purposes (though legally it will still be Novo Nordisk A/S). The Danish drugmaker has a new tagline — Lasting Health Starts Now — and a new logo, as well, with the Apis bull facing right (toward the future, according to Novo) instead of left. “The rebrand is designed to be more memorable and relevant to the way people engage with their health today,” Novo said in a news release.

Additionally, the company introduced a set of four principles, collectively called The Novo Way, that will form the foundation of its corporate culture: customer obsession, competitiveness, clarity, and care and integrity. Tania Sabroe, an executive vice president at the company, said the updated culture and rebrand will sharpen Novo’s competitive edge and strengthen its role as a trusted partner in healthcare.

In separate news, Novo entered into a strategic collaboration and license agreement with Orbis Medicines, a startup that is also based in Denmark, to discover and develop next-generation oral macrocyclic treatments for unspecified cardiometabolic diseases. Orbis could receive up to $1.4 billion in upfront and milestone payments, and Novo agreed to make a strategic investment of an undisclosed amount in Orbis.

According to the announcement, Orbis’ platform integrates generative AI and high-throughput synthesis capabilities to develop synthetic macrocycles optimized for oral bioavailability. These macrocycles have the potential to replace injectable drugs and, Biopharma Dive noted, address limitations associated with standard oral peptides, such as poor digestive absorption.  

Johnson & Johnson is requiring 340B providers to submit claims-level data for the company’s covered outpatient drugs, effective Sept. 15, if they want to continue receiving upfront 340 pricing. The drugmaker notified covered 340B entities of the change in a letter dated Sept. 4. Depending on the type of drug, the required pharmacy or medical claims data is to be submitted within 45 to 60 days of when it is dispensed.

Covered entities that fail to comply with the new policy will receive at least two notifications of noncompliance and a notice of pricing suspension before their access to 340B pricing is suspended, J&J said in the letter. Once they have submitted the data required to bring them back into compliance, 340B pricing will be restored within 10 business days.

J&J said the new policy applies to all covered outpatient drugs “regardless of drug dispensing or administration location, inventory management system, or payer coverage.” In a separate notice, the company advised 340B covered entities that they can only receive 340B pricing at a single contract pharmacy within 40 miles of their parent location.  

Pharmacy care coordination is linked to lower medical costs for cancer patients — as well as fewer visits to emergency departments and hospitalizations, according to a study conducted by researchers at Humana. The researchers analyzed health outcomes and medical costs for Humana Medicare Advantage plan members with Part D prescription plan coverage who received their specialty medications through Humana’s CenterWell Specialty Pharmacy, along with medication adherence care coordination guidance through the pharmacy’s Oncology Center of Excellence.

Over the course of a year, the care coordination services were associated with an 11.7% reduction in hospitalizations, a 2.7% drop in ED visits, and a 7.7% decrease in medical costs due to affiliated pharmacy use. Humana said in a press release the study showed that integrated specialty pharmacy care was associated with faster access to specialty medications (nearly 6 days faster, on average) when compared with nonintegrated specialty pharmacies, and lower annual out-of-pocket costs (approximately $106 per plan member) when compared with those who did not use integrated specialty pharmacies.

Employed physicians reported experiencing worse well-being as compared with independent physicians in a recent survey of 1,000 physicians. For example, more employed physicians reported having inappropriate feelings of anger, tearfulness, or anxiety (62% vs. 54% of independent physicians), experiencing debilitating stress (62% vs. 51%), and feeling hopeless or as if they had no purpose (35% vs. 27%).

Among the employed physicians who reported experiencing distress, the top contributing factors were the pace of work and the expectations placed upon them by the organization they worked for. Among independent physicians who reported experiencing distress, the main contributing factors were administrative burdens and financial pressure. The survey, which was conducted by The Physicians Foundation in collaboration with Medscape, also revealed differences in mental health and well-being among physicians based on age, sex, and geographic practice setting.  

Oracle Health has launched a clinical AI agent for nurses to ease the burden of documentation and streamline inpatient care coordination. Embedded directly into the Oracle Health Foundation electronic health record, the AI agent includes voice-enabled discrete charting, acute nursing summaries, and voice-driven chart navigation, Oracle said in a news release. The company released its clinical AI agent for physicians nearly two years ago.

Providence announced a $1.1 billion gift from Phil and Penny Knight to Providence St. Vincent Medical Center and Providence Heart Institute, referring to it as one of the largest donations to a healthcare institution in U.S. history. The funds will be used to “create Oregon’s first hospital dedicated to women’s health, drive forward the next generation of pioneering cardiovascular treatments at Providence Heart Institute, and advance innovative solutions in prevention, diagnostic, and patient support programs,” Providence said in a press release, acknowledging a longstanding partnership between the Knights, Providence Heart Institute, and the institute’s executive medical director, Dr. Dan Oseran. The Knights have given $200 million to Providence Heart Institute in the past decade, Becker’s Hospital Review reported. Phil Knight is the founder of Nike; Penny is his wife.  

What we’re reading

The Next Decade of Health Spending—Affordability and Value. JAMA, 9.14.26 (subscription required for access)

AI Can Detect Cancer Years Before Symptoms Appear. Health Policy Has Not Caught Up. Health Affairs, 9.14.26

Redesigning Value-Based Payment Models to Support Substance Use Treatment Systems. NEJM Catalyst, 9.16.26 (abstract available; subscription required for access to the full article, which offers substantial detail of the strategy, design, outcomes and more)