Drisapersen이 '획기적인 치료제'로 지정받다

• FDA는 dystrophin에 유전자의 엑손 51의 건너 뛸 수 있는약품인 DMD에서 누락 근육 단백질의 생산으로 이어질 수 있도록 설계한 글락소 스미스 클라인의 실험 Duchenne MD 약물 drisapersen에 "획기적인 치료제"로 지정하였습니다.  

• 획기적인 치료 지정은 증가 FDA의 지침을 제공하고, 특정 기준을 충족하여 임상 의약품에 대한 검토 프로세스를 가속화됨을 의미합니다.

• drisapersen의 임상 시험은 현재진행형 이다.

우리나라도 참여한 임상약품 입니다. 아직 승인이되어 출품된것은 아니지만 비슷한 엑손스키핑 약물(Eteplirsen)보다 앞선 단계인듯합니다.

원문 나갑니다.

Multinational pharmaceutical company GlaxoSmithKline (GSK) has announced that its experimental Duchenne muscular dystrophy (DMD) drug drisapersen has received breakthrough therapy designation from the U.S. Food and Drug Administration.

Drisapersen is one of two experimental DMD drugs currently in clinical trials that works by a mechanism known as exon skipping. It is designed to allow production of the needed dystrophin protein in the muscles of boys with DMD caused by specific mutations and has shown promising results in a phase 2b clinical trial.

(The other DMD exon-skipping drug currently in clinical trials is eteplirsen, from Sarepta Therapeutics. Both drugs target a region of the dystrophin gene known as exon 51.)

About the FDA designation

FDA breakthrough therapy designation is a mechanism designed to expedite the development and review of drugs intended to treat a serious condition and for which preliminary evidence indicates that the drug may demonstrate substantial improvement over available therapy. It's one of several mechanisms the FDA has developed to speed up the review process for certain types of new medications.

A company whose drug is designated a breakthrough therapy gets advice and meetings from the FDA early in the drug development process, involvement of senior FDA staff members and other experts, and the ability to submit parts of the new drug application as they become finalized.

Drisapersen clinical trials are ongoing (see A Snapshot View: Drisapersen and Eteplirsen to learn more).

출처:http://quest.mda.org/news/dmd-drisapersen-receives-breakthrough-therapy-designation

우리나라뉴스:http://www.bosa.co.kr/umap/sub.asp?news_pk=356916