Duchenne Muscular Dystrophy: Givinostat

Baroness Kramer Excerpts

Tuesday 22nd July 2025 Lords Chamber

Asked by

[Baroness Kramer]

To ask His Majesty’s Government what progress has been made in making givinostat available to boys living with Duchenne muscular dystrophy to slow down degeneration, particularly becoming unable to walk.

[Baroness in Waiting/Government Whip (Baroness Blake of Leeds) (Lab)]

NICE is currently evaluating givinostat for the treatment of Duchenne muscular dystrophy to determine whether it should be funded by the NHS, based on the evidence of its costs and benefits. Its independent appraisal committee met on 10 July, and I understand that NICE will issue draft guidance shortly. I hope noble Lords will appreciate that it would not be appropriate for me to speculate on NICE’s draft guidance or to seek to influence the outcome.

[Baroness Kramer (LD)]

My Lords, boys M and H are friends aged 14 and 11, and both have Duchenne muscular dystrophy. Boy M is receiving givinostat, provided by his NHS trust, and expects to preserve his ability to walk and to be eligible for new drugs in the pipeline. Boy H’s NHS trust is not providing him with givinostat. His family fear he will lose his ability to walk before September, and he is unlikely to be eligible for future drugs. Givinostat is cost-free from the manufacturer under the early access programme, but delivery and monitoring costs fall on the NHS. What can the Minister say to boy H and the 400 other boys like him who are losing in the postcode lottery? Will she meet with some of the families?

[Baroness Blake of Leeds (Lab)]

The noble Baroness raises a very interesting point. The drug companies will provide the drugs for free, but it is down to individual trusts to pay the administrative costs. She highlights that one boy in one trust is being supported, while another boy is not. It is a difficult situation; I completely understand that, and my heart goes out to all the young people who suffer from this dreadful disease. It is critical that the trusts work together and look at other practice. It is not for the Government to intervene and force trusts to do as the noble Baroness proposes. However, if they choose to do so, companies can provide funding to enable the clinical administration of their products in advance of the NICE decision. Perhaps the noble Baroness could go back and recommend that as a course of action.