Catalyst Pharmaceuticals, Inc.


Catalyst Pharmaceuticals is a biopharmaceutical company dedicated to developing and commercializing innovative therapies for rare, debilitating neuromuscular and neurological diseases. They focus on bringing life-changing treatments to patients, with a strong commitment to patient care, accessibility, and global expansion through strategic partnerships.

Industries

biotechnology
health-care
health-diagnostics
pharmaceutical

Nr. of Employees

medium (51-250)

Catalyst Pharmaceuticals, Inc.

Coral Gables, Florida, United States, North America


Products

Amifampridine phosphate (oral tablets)

Oral small-molecule potassium-channel blocking agent developed and commercialized for symptomatic improvement of neuromuscular transmission in Lambert-Eaton myasthenic syndrome; supported by randomized clinical trials and regulatory filings.

Vamorolone (oral suspension)

Oral suspension formulation developed for treatment of Duchenne muscular dystrophy in pediatric patients, authorized for North American commercialization through a strategic collaboration.

Perampanel (commercial rights in U.S.)

Commercial rights acquisition and commercialization of an approved antiepileptic drug in multiple strengths and dosage forms for treatment of partial-onset and generalized tonic-clonic seizures in labeled age groups.

CPP-115 (investigational)

Early-stage clinical candidate under development for refractory infantile spasms (and potential other indications), with orphan designation in the U.S. and EU.

Generic vigabatrin development

Development program to create a generic version of an approved anticonvulsant, involving formulation and regulatory filing activities.


Services

Business development and licensing

Negotiation and execution of licensing and sublicensing agreements and strategic partnerships to expand product portfolio and geographic commercialization.

Clinical trial sponsorship and conduct

Sponsorship and operational support for clinical trials (Phase 1–3) including study design, site selection, monitoring and data collection for rare neuromuscular indications.

Commercial launch and patient access programs

End-to-end launch services including payer engagement, patient assistance, and support services to facilitate patient access to therapies.

Investigator-sponsored research funding

Provision of grants and support for investigator-led studies to advance clinical knowledge in targeted rare diseases.

Medical affairs and field medical liaison support

Deployment of Medical Science Liaisons and medical affairs resources to support clinical education, investigator engagement and evidence dissemination.

Compassionate use / expanded access programs

Management of programs providing access to investigational medicines for patients who lack alternatives and meet program criteria.

Expertise Areas

  • Clinical trial management for rare diseases
  • Neuromuscular and neurological therapeutic development
  • Regulatory strategy and submissions (FDA/EMA)
  • CMC and formulation development for oral drugs
  • Show More (4)

Key Technologies

  • Randomized double-blind and withdrawal clinical trial designs
  • Crossover clinical trial methodology
  • Special Protocol Assessment (SPA)-guided trial design
  • GMP-compliant manufacturing
  • Show More (6)

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