The Scottish Medicines Consortium (SMC) has accepted a treatment for an ultra-rare disease that can cause progressive injury to vital organs via damage to the walls of blood vessels and blood clots in its May decisions.

Restricted use of Alexion’s Ultomiris (ravulizumab) has been recommended by the SMC, for the treatment of patients with a body weight of 10kg or above with atypical haemolytic uremic syndrome (aHUS).

Patients in Scotland who are complement inhibitor treatment-naïve or have previously received Alexion’s Soliris (eculizumab) for at least three months, and who have evidence of response to Soliris, are now eligible for the new treatment.

Ultomiris is given every eight weeks, compared to Soliris’ every two weeks, meaning it could reduce the frequency of healthcare visits and alleviate some of the psychological burden of people living with aHUS and their families.

"The SMC decision is excellent news for aHUS patients and their families. Ravulizumab will make their lives much freer and easier whilst providing management of a life-threatening rare disease,” said Len Woodward, director trustee of aHUS alliance Global Action.

In addition to Ultomiris, the SMC has also accepted Pierre Fabre’s Braftovi (encorafenib) for use in combination with Lilly’s Erbitux (cetuximab) for the treatment of advanced colorectal cancer patients with BRAF V600 gene mutation.

In a statement, the SMC said Braftovi plus Erbitux may improve overall survival, as well as delay disease progression and allow patients to maintain their quality of life.

The committee also accepted the rare lymphoma treatment Ledaga (chlormethine hydrochloride), produced by Recordati Rare Diseases and Helsinn Healthcare.

Legada is used to treat mycosis fungoides-type cutaneous T-cell lymphoma, a rare lymphoma type that mainly affects the skin.

It offers an alternative option to phototherapy for these patients, which require frequent hospital visits.

Finally, the SMC recommended GlaxoSmithKline’s Zejula (niraparib) for the treatment of advanced ovarian cancer.

Zejula, which is administered orally, offers patients ‘greater flexibility’ in their treatment, the SMC said in a statement, in addition to potentially fewer side effects coupled with extended progression free survival.