Novartis $2 million gene therapy for rare disorder is world’s most expensive drug

ZURICH/NEW YORK – Swiss drugmaker Novartis on Friday won U.S. approval for its gene therapy Zolgensma for spinal muscular atrophy (SMA), the leading genetic cause of death in infants, and priced the one-time treatment at a record $2.125 million. FILE PHOTO: Logo of Swiss drugmaker Novartis is seen at its branch in Schweizerhalle near Basel, Switzerland, March 29, 2018. REUTERS/Arnd WiegmannThe Food and Drug Administration approved Zolgensma for pediatric patients under age 2 with SMA, including those not yet showing symptoms. The approval covers babies with the deadliest Type 1…

For Anderson family, an early bet on SMA gene therapy

NEW YORK (Reuters) – When Malachi Anderson was diagnosed with a rare and often deadly disease called spinal muscular atrophy (SMA) as an infant nearly four years ago, his parents Tina and Torence had a decision to make. In years past, many of the babies born with the most severe form of SMA, like Malachi, died before their second birthday. Those who survived longer would likely not walk, eat or breathe on their own. By 2015, drugmakers were testing two experimental therapies in clinical trials: a one-time gene therapy treatment…