MHRA approves givinostat for all people with DMD aged six and older

We are overjoyed at the decision by the Medicines and Healthcare products Regulatory Agency (MHRA) to give approval to Duchenne muscular dystrophy (DMD) treatment givinostat for patients aged six years and older.

The MHRA confirm full approval for patients, six years and older, that start treatment when ambulatory (walking), and issue a conditional approval for patients that start treatment when no longer ambulatory (non-walking). Read Jack's blog about the MHRA decision here.

Results from the Italfarmaco Pivotal Phase 3 EPIDYS Study of givinostat in DMD showed that the clinical trial met its primary and secondary endpoints, showing efficacy in slowing down disease progression.

A huge thank you to everyone who has joined us in our campaign. This is a very special day for our community.

Our co-founder, Alex Johnson OBE, said: "With this decision by the MHRA, we are entering a new dawn. A dawn where regulators are demonstrating that where clinical data is compelling, compassion and flexibility can be used to benefit patients."