Rare Diseases Companies
Browse 147 pharmaceutical and biotech companies active in Rare Diseases — each profile screened against live FDA recalls, Warning Letters and Form 483 observations.
147 companies · FDA compliance signals on every profile
- 3P Biopharmaceuticals S.L.
- Ace Pharmaceuticals B.V.
- ACHN Pharmaceuticals Inc.
- Actelion Pharmaceuticals Ltd
- Advanced Biologics Corp
- Aegerion Pharmaceuticals, Inc.
- AGC Biologics Inc.
- ALMB Pharmaceuticals, Inc.
- Alnylam Pharmaceuticals, Inc.
- Amicus Therapeutics, Inc.
- Amryt Pharma plc
- Analona Therapeutics A/S
- Andelyn Biosciences, Inc.
- Angelini Pharma S.p.A.
- ANV Therapeutics Inc.
- ApoPharma Inc.
- Armr Sciences Inc.
- Bachem Holding AG
- Beone Medicines AG
- Bicycle Therapeutics plc
- BillionToOne Inc.
- Biogen Inc.
- BioMed Pharmaceuticals Inc.
- Biotech X Corporation
- Biotherapies Corporation
- Blueprint Medicines Corporation
- BovX Pharmaceuticals Inc.
- Cabaletta Bio
- CatalYm GmbH
- Chai Discovery LLC
- Chiesi Farmaceutici S.p.A.
- Chiesi USA, Inc.
- Chongqing Precision Biotech Co., Ltd
- Chugai Pharmaceutical Co., Ltd.
- Clementia Pharmaceuticals Inc.
- CM Pharmaceuticals Inc.
- Connect Biopharm LLC
- Corstasis Therapeutics Inc.
- Crinetics Pharmaceuticals, Inc.
- Crispr Therapeutics Ag
- Cycle Pharmaceuticals Ltd.
- Daiichi Sankyo Co., Ltd.
- Debiopharm International SA
- Developer Company Inc.
- Dova Pharmaceuticals, a subsidiary of Swedish Orphan Biovitrum AB (Sobi)
- Dragon Boat Biopharmaceutical Co., Ltd.
- Elisigen, Inc.
- Emmaus Life Sciences, Inc.
- Ergomed Plc
- EUSA Pharma
- FHI Clinical, Inc.
- Fondazione Telethon
- Forma Therapeutics Holdings, Inc.
- GeneCure Biotech Inc.
- Genetic Therapies Inc.
- GeneTx Biotherapeutics, LLC
- Genzyme Corporation
- Global Biotechnologies Corporation
- Global Pediatric Research Institute
- GW Pharmaceuticals plc
- Health Innovations Incorporated
- Horizon Therapeutics plc
- Horizon Therapeutics Public Limited Company
- HRA Pharma
- ICON Public Limited Company
- Immatics US, Inc.
- Immedica Pharma AB
- Innovative Biotherapy Corporation
- Innovative Life Sciences Inc.
- International Drug Development Institute
- Ipsen Biopharmaceuticals
- Ipsen S.A.
- Isabella Graziadei Inc.
- JH Pharma Inc.
- Kbio Inc.
- Kiniksa Pharmaceuticals, Ltd.
- Kira Pharmaceuticals
- Leadiant Biosciences, Inc.
- LFB Biotechnologies S.A.
- LFB SA
- Lucane Pharma SA
- Lumos Pharma, Inc.
- Mabgeek Biotechnology Co., Ltd.
- Mallinckrodt Pharmaceuticals
- Medunik Canada Inc.
- Mereo BioPharma Group plc
- Mirum Pharmaceuticals, Inc.
- ModernaTX, Inc.
- NeuroBioPharma Inc.
- NextGen Pharmaceuticals Inc.
- Nova Biopharmaceuticals, Inc.
- Novo Holdings A/S
- NS Pharma, Inc.
- Nycomed Pharma AS
- Ocugen, Inc.
- Orsini - Rare Disease Pharmacy Solutions
- Pfizer İlaçları Limited Şirketi
- PharmaEssentia
- PharmaResearch Inc.
- PharmaXYZ Corporation
- Pharming Group N.V.
- Pharming Technologies B.V.
- Polaris Pharmaceuticals Inc.
- Profile Pharma Ltd.
- Prosensa Therapeutics B.V.
- Protalix BioTherapeutics, Inc.
- Proveca Ltd
- RareCare Pharmaceuticals Inc.
- RareGene Biotech Inc.
- Reata Pharmaceuticals, Inc.
- Recordati S.p.A.
- Regeneron Pharmaceuticals, Inc.
- Rentschler Biopharma SE
- Retrophin, Inc.
- Rigel Pharmaceuticals, Inc.
- RND Pharmaceuticals Inc.
- Rondo Therapeutics, Inc.
- Sanofi Genzyme
- Sanofi S.A.
- Sanofi-Aventis Canada Inc.
- Sanofi-Aventis Singapore Pte Ltd
- Sanofi-Aventis U.S. LLC
- Shire Human Genetic Therapies, Inc.
- Sigma-Tau Pharmaceuticals
- SkyBio Inc.
- SLV Pharmaceuticals Inc.
- SpeciPharma Inc.
- Star Therapeutics Inc.
- SynAct Pharma Aps
- Takeda Pharmaceutical Company Limited
- Takeda Pharmaceuticals America, Inc.
- Takeda Pharmaceuticals Company Limited
- Takeda Pharmaceuticals International AG
- Takeda Pharmaceuticals North America, Inc.
- Takeda Pharmaceuticals U.S.A., Inc.
- TolBiotech Pharmaceuticals Inc.
- Travere Therapeutics, Inc.
- Trogenix ltd
- Unknown
- Vst Bio, Corp.
- Vuessence Inc.
- Xeris Pharmaceuticals, Inc.
- XLT Biopharmaceuticals Inc.
- Y-Trap, Inc.
- Zaffre
- Zambon Company S.P.A.
- Zogenix, Inc.
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