Wednesday, July 8, 2026

Drug profile · INN

risdiplam

risdiplam (EVRYSDI) is a smn2 pre-mrna positive modulator. Associated with Hoffmann-La Roche. Route of administration: oral. Current US regulatory status: approved.

FDA: approved EMA: approved TGA: approved Musculo-skeletal system (M09) M09AX10
US status approved
Patents linked 2
Pipeline programs 4
News articles 1
Drug details — risdiplam
US status
approved
Class
Musculo-skeletal system (M09)
Route
ORAL
Patents
2
Programs
4
Data quality
0.84

Quick answer

risdiplam (EVRYSDI) is a smn2 pre-mrna positive modulator. Associated with Hoffmann-La Roche. Route of administration: oral. Current US regulatory status: approved.

Key facts

INN risdiplam
Brand EVRYSDI
Mechanism SMN2 pre-mRNA positive modulator
Target SMN2 pre-mRNA
Therapeutic class Musculo-skeletal system (M09)
Route ORAL
Formulation FOR SOLUTION
ATC code M09AX10

Mechanism of action

SMN2 pre-mRNA positive modulator

Primary target: SMN2 pre-mRNA

Approval history

Authority Status Date
FDA approved
EMA approved
TGA approved

Recent coverage

Pipeline programs

  1. BN42644 Phase 3 · HOFFMANN-LA ROCHE · Spinal Muscular Atrophy (SMA)
  2. BN44619 Phase 2 · HOFFMANN-LA ROCHE · Spinal Muscular Atrophy (SMA)
  3. BN44620 Approved · HOFFMANN-LA ROCHE · Spinal Muscular Atrophy (SMA)
  4. BN44621 Approved · HOFFMANN-LA ROCHE · Spinal Muscular Atrophy (SMA)

Related & competitor drugs

  1. allopurinol Musculo-skeletal system (M04)
  2. colchicine Musculo-skeletal system (M04)
  3. denosumab Musculo-skeletal system (M05)
  4. eteplirsen Musculo-skeletal system (M09)
  5. febuxostat Musculo-skeletal system (M04)
  6. ibandronic acid Musculo-skeletal system (M05)
  1. US 9969754

    Compounds for treating spinal muscular atrophy

    Expires Mon May 10 2038 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted
  2. US 9586955

    Compounds for treating spinal muscular atrophy

    Expires Mon Mar 02 2037 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

Frequently asked questions

What is risdiplam?

risdiplam (EVRYSDI) is a smn2 pre-mrna positive modulator. Associated with Hoffmann-La Roche. Route of administration: oral. Current US regulatory status: approved.

What is the brand name for risdiplam?

EVRYSDI is a marketed brand name for risdiplam. Check FDA Drugs@FDA for the current US label holder and prescribing information.

What is the US approval status of risdiplam?

risdiplam has approved status in the United States according to our Drugs@FDA-derived records. Verify on the official FDA label before clinical or commercial decisions.