The Scottish Medicines Consortium (SMC) has released its February guidance, advising six new medicines to NHS Scotland.

Among the decisions are the first two medicines assessed within the new “ultra-orphan pathway”, a new approach introduced earlier this year for drugs that are intended for extremely rare conditions.

In the SMC’s latest meeting, Novartis’ Luxturna (voretigene neparvovec) and Kyowa Kirin’s Crysvita (burosumab) were the two new ultra-orphan drugs to be instated by the body, a one-time therapy for an extremely rare type of inherited retinal dystrophy and a treatment for X-linked hypophosphataemia, respectively.

Luxturna has  been shown to improve vision which would have a significant impact on quality of life, making it appropriate for use on NHS Scotland, and Crysvita has been shown to correct bone defects in children, which can result in lifelong benefits.

The decision-making institution says that for these ultra-orphan medicines, companies are now required to provide plans describing how further data on the effects of the medicines, including those on the patient and carer lived experience, will be collected. After that, they will be available on the NHS in Scotland for a period of three years while this information is gathered.

SMC chairman Dr Alan MacDonald said that the new approach will “allow patients with these extremely rare conditions to benefit from access to treatment while companies gather more information on clinical effectiveness.”

He went on to confirm that the “Scottish Government will announce when these medicines are available for prescribing in NHSScotland.”

As for the rest of the approvals, Sanofi got the green light for Libtayo (cemiplimab) - subject to ongoing evaluation and future reassessment by SMC. The drug is used to treat advanced cutaneous squamous cell carcinoma (CSCC) in patients who are not candidates for curative surgery or curative radiation, and was considered through SMC’s Patient and Clinician Engagement (PACE) process, which is used for medicines to treat end of life and very rare conditions.

Also accepted through PACE was Takeda’s Revestive (teduglutide), the only licensed medicine for the treatment of short bowel syndrome (SBS), and Braftovi (encorafenib), Array Pharma’s treatment for certain patients with metastatic melanoma when used in combo with Mektovi (binimetinib).

Finally, the committee said it was unable to accept AstraZeneca’s Lokelma (sodium zirconium cyclosilicate) for the treatment of hyperkalaemia - high levels of potassium in the blood - as the company’s evidence about the economic benefits of the medicine was “not strong enough.”

AZ has rebutted the decision, saying it is "disappointed" with the decision as "people with hyperkalaemia have seen almost no treatment innovation in nearly 50 years."

In a statement, the company went on to say that it is "addressing the questions from the SMC and will be resubmitting Lokelma for reconsideration later this year.

"Approximately 5,000 people in Scotland are affected by hyperkalaemia, and AstraZeneca remains committed to enabling Scottish patients to access additional treatment options for the condition."

The full list of decisions was published by the SMC on February 10th 2020.