England’s NICE Says No To Santhera’s DMD Drug Agamree Due To ‘Unreliable Modeling’

Santhera Pharmaceuticals did not provide enough evidence to demonstrate that its Duchenne muscular dystrophy drug Agamree was a cost-effective use of resources, according to draft guidance from England’s health technology assessment body, NICE.

DMD

NICE, the organization that makes reimbursement recommendations for medicines used on the National Health Service in England and Wales, has provisionally turned down Santhera Pharmaceuticals’ Agamree (vamorolone) for funding, less than three months after the dissociative steroid was granted approval by the UK regulator, the MHRA.

Agamree, which was authorized by the MHRA to treat children aged four years and older with Duchenne muscular dystrophy (DMD) on 12 January, was positioned by Switzerland-based Santhera as a drug that offered far fewer side effects than

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