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Vertex to Request Review of Orkambi, Symkevi Plus Kalydeco for Public Funding in Scotland

Vertex Pharmaceuticals has announced its intention to submit an application to the Scottish Medicines Consortium (SMC) to review the clinical and cost-effectiveness of the company’s cystic fibrosis (CF) medicines, Orkambi (lumacaftor/ivacaftor), as well as Symkevi (tezacaftor/ivacaftor) in combination with Kalydeco (ivacaftor).

Following the appraisal of data submitted by Vertex, the SMC will decide whether to accept these CF therapies for use on the National Health Service (NHS) in Scotland, the country’s publicly funded healthcare system.

“We are hopeful that, through this process, all eligible patients in Scotland could have access to our medicines soon,” Ludovic Fenaux, senior vice president at Vertex International, said in a press release.

If approved, these therapies could become available in Scotland this year.

Medications under review by the SMC and not yet available on the NHS may be obtained by clinicians through the Peer Approved Clinical System Tier 2(PACS Tier 2). During the appraisal process of Vertex’s submission, clinicians can request access to these treatments through PACS Tier 2 on a case-by-case basis until a final decision is made.

Moreover, as per the agreement between Vertex and the Scottish government, approved PACS Tier 2 requests will be eligible for a price discount. The discount amount is currently undisclosed.

If the SMC decides to recommend Orkambi, Scotland will join other countries that made this therapy available to CF patients; including the U.S., Australia, the Netherlands, Denmark, Germany, Italy, the Republic of Ireland, Austria, and Sweden.

Symkevi in combination with Kalydeco was recently approved in the European Union for CF patients, ages 12 years or older who carry F508del mutations. Symkevi is approved and marketed in the U.S. as Symdeko.

Vertex’s decision to submit these therapies for review by the SMC comes after detailed discussions between the company and the Scottish government. Medicines that treat a life-threatening condition such as CF, and those with orphan medicine status from the European Medicines Agency are evaluated by the SMC through its orphan medicines process.

“Our recent conversations with the Scottish Government and the SMC’s orphan medicines process have provided important flexibility for evaluating precision medicines, such as cystic fibrosis transmembrane conductance regulator (CFTR) modulators. Their methods reflect the innovative nature of medicines that have the potential to extend life for patients with rare diseases, like CF,” Fenaux said.

CF is estimated to affect 900 people in Scotland.

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