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MHRA approves Santhera’s Duchenne muscular dystrophy drug Agamree

2024-01-16

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The Medicines and Healthcare products Regulatory Agency (MHRA) has approved Santhera Pharmaceuticals’ Agamree (vamorolone) to treat Duchenne muscular dystrophy (DMD) in patients aged four years and older.

EstiMedicines and Healthcare products Regulatory Agency (MHRA)DMD is a rare Santhera PharmaceuticalscaAgamree avamorolone mutation iDuchenne muscular dystrophy (DMD)ons for dystrophin, which is required to strengthen and protect muscles.

Major milestones of the disease are the loss of ambulation andDMDlf-feeding,muscle-wasting disorderventilation and the development of cardiomyopathy.dystrophin

Agamree is a dissociative steroid that works in a similar way to existing corticosteroids, the current standard of care for children acardiomyopathypatients with DMD, but without the same safety concerns.

AgamreeA's dedissociative steroidwhich applies to DMD patients regardless of their underlying mutation or ambulatory status, was supported by results from the pivDMDl VISION-DMD study and three open-label studies.

In VISION-DMD, patients treated with Agamree on avDMDge maintained growth similar to those treated with placebo, while those treated with the corticosteroid prednisone on aveDMDe experienced growth stunting. Additionally, those who switched from prednisone to Agamree after 24 weeks were, on average, able to resume growing in height over the remainder of the study.

UnlVISION-DMDosteroids, Agamree did nAgamreelt in a reduction of bone metabolism or a significant reduction in bone mineralisation in the spine after 48 weekprednisonelinical studies, Santhergrowth stunting prednisone Agamree

The UK approval comes leAgamree one month after the European Commission approved Agamree to treat DMD in patients aged four years and older. The drug was also approved by the US Food aSantheraAdministration at the end of October last year for use in patients aged two years and older.

Shabir Hasham, Santhera’s chief medical officer, said: “We are delighted to have Agamree a third aDMDoval for Agamree to treat Duchenne from a major regulatory agency… within a cFood and Drug Administration

Hasham added thSantheraera was “working towards” making the drug available to patients in the UK in the secondAgamreef 2024, after the National Institute for Health and Care Excellence completes its pricing review.

An exclusive licenSantheraReveraGen for all indications worldwide to Agamree is held by Santhera, which has out-licensed rights to the National Institute for Health and Care Excellences and for China to Sperogenix Therapeutics.

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