A new treatment option for the very rare condition infantile onset 5q spinal muscular atrophy (type I SMA) is among five medicines that have been endorsed by Scottish cost regulators for use on the NHS.
Biogen Idec’s Spinraza (nusinersen) was accepted by the Scottish Medicines Consortium to treat the condition, which results in devastating muscle wasting that can cause patients to lose the ability to move, breathe and swallow.
In type 1 SMA, symptoms usually start shortly after birth, and affected children rarely survive beyond two years of age. The committee did not recommend the therapy for type 2 and 3 SMA.
Spinraza is an antisense oligonucleotide (ASO) designed to treat SMA caused by mutations or deletions in the SMN1 gene located in chromosome 5q that leads to SMN protein deficiency.
The therapy was cleared for use in Europe in following accelerated assessment in May 2017, based on data from two pivotal multicenter, controlled studies which showed clinically meaningful efficacy and a favourable benefit-risk profile, the company previously said.
Elsewhere, Merck and Pfizer’s Bavencio (avelumab) was accepted for the treatment of Merkel cell carcinoma (MCC), an aggressive, rare form of skin cancer.
The drug is a form of immunotherapy that works by harnessing the power of the patient’s own immune system to destroy their cancer cells, approved in September last year as the first and only targeted systemic treatment to be licensed for metastatic MCC.
Data from the Phase II trial JAVELIN Merkel 200 showed durable responses to drug; the proportion of patients who achieved an objective response was 28 (31.8 percent) of 88 patients, including eight complete responses and 20 partial responses.
Funding for Bayer’s Stivarga (regorafenib) was cleared for the treatment of advanced liver cancer in patients who have already undergone previous treatment with sorafenib.
The Committee noted that in a Phase III study involving patients with hepatocellular cancer that had progressed on sorafenib treatment, the drug significantly improved overall survival compared with placebo on a background of best supportive care.
Also accepted following a resubmission was Actelion’s Uptravi (selexipag) for pulmonary arterial hypertension (PAH, abnormally high blood pressure in the arteries of the lungs).
However, the SMC has restricted the drug’s use to combination therapy in a sub-population of patients with PAH specifically those in WHO FC III who are insufficiently controlled with an ERA and a PDE-5 inhibitor and who would be considered for treatment with inhaled iloprost.
The SMC Committee also waved through LEO Pharma’s novel biologic Kyntheum (brodalumab) for the treatment of severe plaque psoriasis.
By binding to this specific receptor on the cells of the skin, the drug blocks the biological activity of several pro-inflammatory IL-17 cytokines involved in plaque formation, offering a different mechanism of action to all other psoriasis biologics currently available, which target free inflammatory mediators.
In the clinical trials, 37-44 percent of patients treated with the drug achieved complete skin clearance (PASI 100) at week 12, compared with 19-22 percent with ustekinumab, with “high levels” of skin clearance sustained with continuous brodalumab treatment through week 52.