Agamree recommended for NHS use in UK, approved in China
Oral treatment to maximize benefits of corticosteroids with reduced side effects
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Agamree (vamorolone) has been recommended for use in the National Health Service (NHS) in England, Wales, and Northern Ireland for treating Duchenne muscular dystrophy (DMD) patients, 4 and older.
The recommendation came from the U.K.’s National Institute for Health and Care Excellence (NICE) and follows Agamree’s approval in the U.K. early this year. Agamree, a so-called dissociative steroid treatment, was developed by Santhera Pharmaceuticals, which licensed rights to the therapy for North America to Catalyst Pharmaceuticals and for China to Sperogenix Therapeutics.
“We are delighted that NICE has recommended Agamree for patients with DMD, and we are committed to working closely with the NHS to ensure patients can access Agamree as quickly as possible,” Dario Eklund, CEO of Santhera, said in a company press release.
Recommendation expected to take effect within 90 days
The recommendation is expected to take effect within 90 days, making Agamree funded and available to patients in U.K. regions. To expand access to Scotland, reimbursement processes are also underway with the Scottish Medicines Consortium.
“We are very proud that a treatment that we have supported for so long will now be available in the NHS,” said Emily Reuben, CEO of Duchenne UK.
Michela Guglieri, MD, a consultant neurologist at Newcastle Hospitals NHS Foundation Trust, added: “Agamree represents a significant advancement for the treatment of DMD, offering patients an alternative option to slow down the progression of the disease while reducing some of the side effects of traditional corticosteroids that affect patient’s quality of life.”
Elsewhere, Agamree was approved in China, making it the country’s first treatment for DMD.
“The approval of Agamree through the Priority Review Program reflects the government’s high level of attention to the development of rare disease drugs,” said Yan Zhiyu, chairman and CEO of Sperogenix, in a separate press release.
DMD affects more than 70,000 families in China, “and until now, there had been no approved treatment option for patients,” Eklund said.
The most common type of muscular dystrophy, DMD is characterized by progressive muscle weakness and wasting, and more commonly affects boys than girls.
Corticosteroids are a type of medication often used to reduce inflammation in the body and are the standard treatment for DMD. However, when used long term, they can cause significant side effects, such as stunted growth, fragile bones, and weight gain.
Agamree designed to preserve muscle function, ease inflammation in DMD
Agamree is designed to preserve muscle function and ease inflammation in DMD, and to maximize the benefits of corticosteroids while reducing the side effects commonly associated with them.
Clinical trial data support Agamree’s efficacy and safety. In the pivotal VISION-DMD Phase 2b trial (NCT03439670), Agamree treatment resulted in similar effectiveness in preserving muscle function but fewer side effects (particularly those related to bone health, growth, and behavior) compared to the standard steroid prednisone.
VISION-DMD enrolled 121 boys with DMD who could walk, ages 4-6, assigned to receive oral Agamree (2 or 6 mg/kg), prednisone (0.75 mg/kg), or a placebo once daily for six months. Then, all received Agamree for another six months.
Agamree is the first medication for DMD to receive full approval in the U.S., the European Union, the U.K., and China.
“The approval of vamorolone (Agamree) is the culmination of a global effort of scientists, clinicians and patient advocacy groups investing in and supporting the development of vamorolone,” Reuben said.
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