Risdiplam bulk supplier for pharma manufacturers

Risdiplam 0.75 mg/mL Suppliers & Bulk Manufacturers

Available Forms: Oral Solution

Available Strengths: 0.75 mg/mL

Reference Brands: Evrysdi (USA/EU)

Category: Neurology

Risdiplam is an oral survival motor neuron 2 (SMN2) splicing modifier used to treat spinal muscular atrophy (SMA) in adults and children aged 2 months and older. It works by increasing the production of functional SMN protein, essential for maintaining motor neuron function. Risdiplam helps improve muscle strength and slow disease progression in SMA patients.

Ask About Access

Usually answered within 48 hours | GMP documents shared before you commit

Manufacture or export Risdiplam? List it or send an offer →

Product Description:

Risdiplam is a small-molecule splicing modifier for spinal muscular atrophy. In SMA the SMN1 gene is lost, and the backup SMN2 gene produces mostly truncated protein because exon 7 is usually skipped during splicing. Risdiplam binds the SMN2 pre-mRNA and promotes inclusion of exon 7, increasing full-length, functional SMN protein throughout the body. It was the first oral treatment for SMA, and because it distributes systemically it also reaches tissues outside the central nervous system.

It is taken once daily, dosed by age and weight, from birth onwards. The alternatives are intrathecal nusinersen, which needs repeated lumbar punctures, and one-time gene therapy, so an oral daily option has practical value for many families and older patients. Common adverse effects include fever, diarrhoea and rash. Animal studies showed effects on male fertility, and effective contraception is needed because of embryo-fetal toxicity in animals.

Sourcing notes. Originator product, on patent, no generic. Originally a powder reconstituted to oral solution by the pharmacist, which then needs refrigeration and has a limited in-use shelf life; a tablet presentation has since been approved in some markets. Very high annual cost makes access programme and reimbursement status the practical constraint. Supply is through authorised distribution.

How Risdiplam is supplied today, and licensing ahead of patent expiry

Risdiplam is a patented innovator medicine. Where its patents are in force, which today includes most major markets, it can only be supplied by the originator or its licensees, through the routes below. Patents do expire, though, and generic development and licensing deals for post-patent markets start years in advance. If you are developing Risdiplam, hold a dossier for it or want to license it, talk to us.

Named-patient import Where a patient needs the product in a market where it is not registered, named-patient import is the lawful route. It requires the treating physician's documentation and authorisation from the destination regulator. We handle this pathway. Named-patient access

Authorised distribution For registered markets, supply runs through the manufacturer's authorised distribution channel. We can identify the authorised route for your market and tell you whether the product is registered there at all. Innovator sourcing

A genericised alternative Where the clinical requirement is the drug class rather than this specific molecule, there is often an off-patent alternative that is faster to register and materially cheaper. Tell us the indication and target market and we will say plainly whether one exists. Discuss alternatives

Licensing and generic development Patents expire country by country, and in many countries development and dossier filing can start before expiry under research and regulatory-review exemptions. We connect manufacturers developing this molecule, dossier holders, licensees and tech-transfer partners for markets where it is, or will become, off-patent. Partner on this molecule

Ask about Risdiplam supply →

Back to Listing

Frequently Asked Questions

Risdiplam is used for the treatment of spinal muscular atrophy (SMA) in patients two months of age and older.

Risdiplam is made from a small-molecule splicing modifier that increases the production of functional survival motor neuron (SMN) protein in the body.

The trade name of Risdiplam is Evrysdi.

Risdiplam is made by Genentech, a member of the Roche Group, in collaboration with PTC Therapeutics and the SMA Foundation.

The generic name of the product is Risdiplam.

The brand name of Risdiplam is Evrysdi.

Risdiplam is manufactured by Genentech, Inc. in the United States.

Evrysdi (Risdiplam) is developed and sold by Roche (developed with PTC Therapeutics). PharmaTradz handles enquiries for authorised supply of the originator product and for named-patient import where it is not registered.