The new drug Zolgensma of Novartis has lately been approved for marketing by the U.S. FDA to treat pediatric patients with spinal muscular atrophy (SMA). SMA is a rare disease (orphan disease), with the incidence of only 1-2/100,000. It's worth mentioning that as gene therapy, Zolgensma is priced at USD2.125 million/time which is sky-high. This causes people to think about the choice of rare disease drugs: for patients, it is a question as to whether to choose biologics (expensive) or small molecule drugs (cheap); for pharmaceutical practitioners, choosing which drugs for development is also a question for thought.
