Press release

March 18, 2020

Risdiplam approaching mid-2020 PDUFA date 

This will be the third, high cost drug treatment option emerging for spinal muscular atrophy (SMA) in less than five years; many more are in the pipeline.1

Drug name: Risdiplam 

Manufacturer: Roche/PTC Therapeutics

ROA: Oral  

Condition: Treatment of SMA; all types, all ages 

Condition overview: 

Spinal muscular atrophy (SMA) is a rare, genetic disease. It leads to the progressive loss of spinal cord nerve cells that control muscle movement. Depending on the type of SMA contracted, an individual’s physical strength, and ability to walk, eat or breathe can be significantly diminished or lost.

SMA affects approximately one in 11,000 babies. About one in every 50 Americans is a genetic carrier. SMA can affect any race or gender but occurs more commonly in males.2 As many as 10,000 to 25,000 children and adults in the United States currently have SMA.

SMA is divided into subtypes, based on age of onset and motor function milestone achieved. Type I is usually diagnosed during an infant’s first six months and is the most severe and most common. SMA types II, III and IV, are diagnosed after 6 months and are less severe.

Current treatments

  • Biogen’s Spinraza® (nusinersen) was launched first, in 2016. It is an intrathecally administered antisense oligonucleotide for the treatment of SMA in pediatric and adult patients. Spinraza use has been primarily in older patients with SMA. 
  • Zolgensma® (onasemnogene abeparvovec-xioi) is an intravenous one-time gene therapy for pediatric patients less than two years of age with SMA Type I.3  Zolgensma replaces the missing or defective gene with a functional copy. Zolgensma’s focus is primarily newly-diagnosed infants. A look at Spinraza and Zolgensma.
  • As a new treatment, Risdiplam may capture patients who have elected not to use, or who have stopped taking, Spinraza. If approved, Risdiplam, an orally administered liquid, would be the first at-home administered medicine for people living with SMA.

PDFUA date: May 24, 2020

Benefit: Risdiplam will impact the pharmacy benefit because it is taken orally

Prime monitors the drug pipeline

The drug pipeline is full of new, groundbreaking specialty drugs that may help members feel better and live well. There are more than 20 drugs in the SMA pipeline, three of which are in Phase 2 studies.4-6  [FDA review process

Prime focuses on developing clinical strategies designed to keep clients ahead of drug trends — because it’s easier to manage change when you see it coming. 


1. Curesma. SMA Drug Pipeline. Accessed in February 2020 at:

2. What is SMA? Cure SMA. (n.d.). Retrieved Oct. 16, 2018, from

3. Types of SMA. Retrieved Nov. 2018, from

4. Roche. Roche presents data from the risdiplam pivotal FIREFISH and SUNFISH studies in spinal muscular atrophy at the 2019 AAN Annual Meeting. Accessed in February 2020 at:

5. Roche. Roche’s risdiplam meets primary endpoint in pivotal FIREFISH trial in infants with type I spinal muscular atrophy. Accessed in February 2020 at: 

6. SMA Foundation. 2019: SMA drugs approved and in clinical trials. Accessed February 2020 at: 

About Prime Therapeutics

Prime Therapeutics LLC (Prime) makes health care work better by helping people get the medicine they need to feel better and live well. Prime provides total drug management solutions for health plans, employers, and government programs including Medicare and Medicaid. The company processes claims and offers clinical services for people with complex medical conditions. Prime serves more than 28 million people. It is collectively owned by 18 Blue Cross and Blue Shield Plans, subsidiaries or affiliates of those plans.

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