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Poland to reimburse world’s most expensive SMA drug

Tomasz Gzell/PAP

Poland will reimburse a genetic therapy dubbed “the most expensive drug in the world”, which is used to treat spinal muscular atrophy (SMA).

Adam Niedzielski, the health minister, announced the decision on Monday.

SMA is a rare genetic affecting the central nervous system, peripheral nervous system, and voluntary muscle movement (skeletal muscles).

As a result, there is a gradual atrophy and weakening of the muscles and, consequently, paralysis of the skeletal and respiratory muscles. Without proper treatment, the disease can lead to death from respiratory failure before a child reaches the age of two.

The drug, Zolgensma, used to treat the condition, is known to be one of the most expensive in the world. In an article published in December last year, Forbes magazine said a one-dose treatment cost USD 2.1 million.

“The September reimbursement list will include the most expensive drug against SMA, in terms of a single-dose, namely Zolgensma, which will be intended for children up to six months of age,” Niedzielski told reporters.

“It will, of course, be administered after a screening programme,” he added.

“This is the day when we can say that Poland has joined the group of world’s most advanced countries as far as the comprehensive treatment of SMA is concerned,” Niedzielski stated.

Niedzielski also said that most innovative drugs in Poland were financed by the Medical Fund.

The Medical Fund, which is a new tool in the Polish health care system, was launched in November 2020 on the initiative of President Andrzej Duda.

Niedzielski also said that, as part of the programme to combat SMA, Poland is also introducing another drug, Evrysdi. 

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