NHS Scotland Approves First Ultra-orphan Medicines

The Scottish Medicines Consortium (SMC) has approved ultra-orphan treatments for two rare conditions.

These are the first approvals under the SMC's ultra-orphan pathway, a new approach that has only recently been introduced.

Voretigene neparvovec (Luxturna, Novartis) is a one-off treatment for inherited retinal dystrophy caused by a mutation in the RPE65 gene.

Previously there was no available treatment.

The SMC said data show benefits at least 4 years after treatment.

Burosumab (Crysvita, Kyowa Kirin) is a treatment for X-linked hypophosphataemia that has been shown to correct bone defects in children, which could provide lifelong benefits.

In a statement, SMC Chair Dr Alan MacDonald said: "Voretigene neparvovec and burosumab will now move to the next stage of the ultra-orphan pathway. This new approach will allow patients with these extremely rare conditions to benefit from access to treatment while companies gather more information on clinical effectiveness. [The] Scottish Government will announce when these medicines are available for prescribing in NHS Scotland."

Other Decisions

Cemiplimab (Libtayo, Sanofi) was accepted for interim use for advanced cutaneous squamous cell carcinoma (CSCC) in patients who are not candidates for surgery or radiotherapy.

This decision followed referral to the Patient and Clinician Engagement (PACE) process for rare conditions and end of life care.

Dr MacDonald said that for patients, "our decision to accept cemiplimab on an interim basis means they will be able to be treated while more mature data are gathered on the impact of this medicine."

Teduglutide (Revestive, Shire) was approved for treating adults with short bowel syndrome, also after PACE referral. Teduglutide can improve quality of life by reducing the level of parenteral nutrition required.

Encorafenib (Braftovi, Pierre Fabre) was accepted under PACE for certain patients with metastatic melanoma and a mutation in the BRAF gene, to be used in combination with binimetinib (Mektovi, Pierre Fabre).

Dr MacDonald said: "PACE participants for encorafenib told us patients with advanced melanoma face a poor prognosis, so our decision on this medicine will increase patient choice, which we know will be welcomed."

Not Approved

Sodium zirconium cyclosilicate (Lokelma, AstraZeneca) for hyperkalaemia did not get SMC approval.

Dr MacDonald said: "The company’s evidence around the medicine’s benefits was not strong enough."

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Cite this: Nicky Broyd. NHS Scotland Approves First Ultra-orphan Medicines - Medscape - Feb 10, 2020.

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