According to reports, shortly after being blocked by the pharmaceutical giant AstraZeneca, the rare disease drug maker Sobi won a regulatory victory. The company's rare disease drug Aspaveli received the support of the British regulatory agency.

A few days ago, the National Institute of Health and Care Excellence (NICE) expressed support for Sobi’s paroxysmal nocturnal hemoglobinuria (PNH) drug Aspaveli.

Sobi said in a press release that NICE issued the relevant decision after a quick evaluation of Aspaveli. NICE has recommended Aspaveli as an option for adult PNH patients, which is suitable for patients who have developed anemia after receiving C5 inhibitors (such as AstraZeneca’s Ultomiris) for at least three months. This recommended decision is expected to help Sobi rare Disease drugs will pass the review of the UK drug regulatory agency in the future.

Aspaveli has been marketed in the United States by Apellis under the name Empaveli, and the drug is currently under review by the British Medicines and Healthcare Products Regulatory Agency (MHRA). However, NICE often refuses to accept drug applications due to drug pricing issues. Although the drug may be expensive, Sobi's Aspaveli may have different results. According to data from Drugs.com, the cost of supplying Empaveli in Apellis in the United States is approximately US$36,790 per 160 ml.

Sobi said that PNH is a rare chronic blood disease characterized by the destruction of healthy oxygen-carrying red blood cells. Patients may develop blood clots (blood clots), causing organ damage and even premature death. This disease affects about 900 people in the UK. Patients need frequent blood transfusions after the illness, and cause symptoms such as severe fatigue and anemia. If left untreated, the 10-year mortality rate for this disease is 29%.

At the same time, AstraZeneca's Ultomiris, a drug with the same indication, was also supported by NICE in April this year after offering a confidential discount. The drug can be used in patients with high disease activity who have remained stable on Soliris therapy for at least six months. The drug is administered according to the patient's body weight, but it costs about £50,000 every other month before the discount. It is worth noting that Soliris is also a star product under Alexion. The drug is one of the world’s best-selling rare disease drugs. Its sales in 2020 reached US$4.065 billion. As a long-acting version of the drug, Ultomiris will be released in 2020. The sales have reached 1.077 billion US dollars.