Najdroższy lek na świecie

Amerykańska Agencja Żywności i Leków (FDA) oficjalnie zatwierdziła terapię rzadkiej choroby genetycznej, tzw. rdzeniowego zaniku mięśni kręgosłupa (SMA, ang. spinal muscular atrophy). Jedna dawka leku pod nazwą "Zolgensma", produkowanego przez szwajcarską firmę farmaceutyczną „Novartis”, kosztuje 2,1 mln dolarów, co czyni go najdroższym lekiem świata.​

Producent leku mówi, że ta jedna dawka może uratować życie wszystkim, którzy będą mieć szczęście go dostać.

Trzyletni Donovan Weisgarber jest jednym z chorych na SMA. Gdy się urodził, był zupełnie zdrowy, ale po kilku tygodniach było jasne, że coś jest nie w porządku.

- Miał miesiąc, gdy pojawiły się pierwsze symptomy, mówi matka Donovana Laura Weisgarber z Columbus w Ohio.

Donovan stał się nerwowy, przestał się wiercić i słabł. Okazało się, że cierpi na rdzeniowy zanik mięśni kręgosłupa, wywołany uszkodzonym genem. Choroba niszczy nerwy kontrolujące mięśnie, a dziecko z najcięższą jej postacią żyje około 2 lat.

- Byliśmy zdesperowani. To był zdecydowanie najcięższy okres w naszym życiu, mówi Laura Weisgarber.

Wtedy lekarze zaproponowali rodzicom eksperymentalną terapię genetyczną. Chłopcu wstrzyknięto genetycznie zmienione wirusy będące nośnikami zdrowych kopii genu, którego mu brakowało.

Stan chorego powoli zaczął się poprawiać. Wprawdzie dalej porusza się na wózku inwalidzkim i jest żywiony przez sondę, ale jest już o wiele lepiej.

- Lubi być poza domem, najchętniej w ogrodzie. Może wykonywać wiele normalnych czynności. To cud, cieszy się mama.

Po udanych testach lek, opracowany przez firmę "AveXis" z Bannockburn (Illinois), przeszedł na własność giganta farmaceutycznego "Novartis: i w miniony piątek został oficjalnie zarejestrowany w Stanach Zjednoczonych.

"Novartis" spodziewa się, że "Zolgensma” zostanie jeszcze w tym roku zarejestrowany w Europie i Japonii.

 

Autor: 
M.A./Tłum. Emilia Liberda
Źródło: 

npr.org

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