An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
This expanded access program (EAP) will provide access to risdiplam for eligible participants
 with Type 1 or Type 2 spinal muscular atrophy (SMA) before it is commercially available in
 the United States for the indication of SMA.
Inclusion Criteria:

 All Participants:

 -  Not eligible for treatment with currently approved treatments for SMA, or cannot
 continue treatment with currently approved medications as documented by the treating
 physician, or in the treating physician's judgment, the participant is at risk of
 lack/loss of treatment efficacy of the current therapy.

 -  The participant does not qualify for and has no access to SMA treatment in the context
 of an ongoing clinical trial.

 -  Adequately recovered from any acute illness at the time of screening, and considered
 clinically well enough to participate, in the opinion of the treating physician.

 -  Participants with retinopathy of prematurity should have evidence of stable disease.

 Type 1 SMA Participants:

 - Confirmed diagnosis of 5q-autosomal recessive SMA.

 Type 2 SMA Participants:

 -  Confirmed diagnosis of 5q-autosomal recessive SMA.

 -  Negative blood pregnancy test at screening (all women of childbearing potential,
 including those who have had a tubal ligation), and agreement to comply with measures
 to prevent pregnancy and restrictions on egg and sperm donation.

 -  Males with female partners of reproductive potential must agree to use highly
 effective contraception during therapy, and for at least 4 months after treatment
 discontinuation.

 Exclusion Criteria:

 -  Inability to meet program requirements.

 -  Concomitant or previous participation in any investigational drug or device study
 within 90 days prior to screening or 5 half-lives, whichever is longer.

 -  Administration of other SMN-2 targeting therapy within 120 days of starting risdiplam
 therapy.

 -  Administration of SMA gene therapy within the last 3 months (12 weeks) of receiving
 risdiplam therapy.

 -  Any serious medical condition, treatment, or abnormality in clinical laboratory tests
 that, in the treating physician's judgment, precludes the participant's safe
 participation in the program.

 -  Ascertained or presumptive hypersensitivity (e.g., anaphylactic reaction) to risdiplam
 or to the constituents of its formulation.

 -  Suspicion of illicit drug or alcohol abuse, in the treating physician's judgment.

 -  Any prior use of an inhibitor or inducer of flavin-containing monooxygenases 1 (FMO1)
 or flavin-containing monooxygenases 3 (FMO3) taken within 2 weeks (or within 5 times
 the elimination half-life, whichever is longer) prior to dosing.