Rare Disease Treatments Cleared For Coverage on NHS Scotland

According to a story from Pharma Times, four new medications were recently approved for coverage on the Scottish NHS by the Scottish Medicines Consortium (SMC). Some of these drugs are designed to treat rare diseases such as urea cycle disorders, a rare type of lung cancer, hereditary angioedema, and ovarian cancer. With these approvals, patients in Scotland will have access to new and affordable treatment options for their diseases.


Zeluja was cleared for use in treating patients with advanced ovarian cancer. However, it is worth noting that it is not permitted for use in those patients with BRCA mutations. This therapy was first approved for use in the EU in November of last year. In this approval, the drug was cleared for all patients whether they had the BRCA mutation or not.


Alecensa was approved to treat an unusual type of lung cancer known as ALK-positive non-small cell lung cancer. This type of cancer is dangerous as it is often discovered once it has reached an advanced stage. There are few available treatments for it, but Alecensa has the potential to significantly delay progression of this cancer type. It also offers better outcomes in comparison to the current treatment standard.


The drug Ravicti was admitted to NHS Scotland as a type of long term treatment for urea cycle disorders (UCD) for patients aged two months or older. This disorder prevents the body from getting rid of excess nitrogen, which can lead to a build up of ammonia in the body. This can cause coma, brain damage, and death without treatment. Ravicti is able to reduce ammonia levels in the body.


Ruconest was approved for treating hereditary angioedema, a rare disorder which can cause spontaneous episodes of acute swelling, which can be painful, and, when the airway is involved, potentially fatal. This therapy is meant to help provide rapid relief during attacks, and adds another treatment approach for patients affected by this rare disease.

The approvals were good news for patients that are in need of new treatment approaches. Unfortunately, the  SMC also rejected two medicines which were meant to treat phenylketonuria and hyperkalemia.

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