Evolving Strategies for Rare-Disease Drug Development
Decision Resources, Inc, July 2011
So compelling is the commercial opportunity presented by rare diseases that Novartis has re-engineered its R&D approach to assess these opportunities alongside the proven opportunities offered by more common diseases. Novartis is not alone. Facing looming patent expiries for several blockbusters, drug developers are looking to rare diseases as an important opportunity to recoup anticipated revenue losses. Alongside traditional product licensing agreements and company acquisitions, developers are using a host of other strategies to gain traction in rare diseases, including the formation of dedicated R&D centers and novel approaches to repurposing drugs. Additionally, developers have at their disposal an increasing number of attractive resources, such as the FDA’s Rare Disease Repurposing Database, created through government-driven initiatives, that will further drive rare-disease drug development.
Questions answered in this report:
- In developing drugs for rare diseases, companies have adopted a broad range of strategies. What have been the most commonly used approaches? Which offer the greatest potential for future success in this arena?
- Several government-driven initiatives have been launched in the United States to promote the development of new drugs for rare diseases. What resources are available to developers that seek to enter or grow their presence in rare diseases? How can companies leverage current initiatives to identify and develop commercially and clinically compelling drugs for rare diseases?
- The repurposing of currently approved drugs for rare diseases can give companies an expedited route to market. What are the advantages of repurposing a drug for a rare disease? Are there any limitations to this approach? How can companies best harness the information contained within the FDA’s Rare Disease Repurposing Database to drive product development? Have any companies succeeded in repurposing a current therapy for a rare disease?
- Several major pharmaceutical companies are working to develop drugs for rare diseases by forming commercial partnerships with specialist biotechnology companies and establishing dedicated rare-disease centers. Which rare diseases are companies targeting, and what are the recent alliances that have been established?
Scope of the report:
- New government initiatives to promote rare-disease drug development: The National Institutes of Health’s Therapeutics for Rare and Neglected Diseases Program and National Center for Advancing Translational Sciences and the FDA’s Rare Disease Repurposing Database.
- Corporate initiatives to create rare-disease R&D centers: GlaxoSmithKline’s Rare Diseases, Pfizer’s Rare Disease Research Unit, and Novartis’s Institute for Biomedical Research.
- Rare-disease specialty companies: Amicus Therapeutics, Ultragenyx Pharmaceutical, Bluebird Bio, Synageva BioPharma, and Zacharon Pharmaceuticals.
- Strategies to develop drugs for rare diseases: leveraging government-driven resources, drug repurposing, forming public-private partnerships, and acquiring the rights to novel therapies.
- Commercial agreements between major pharmaceutical companies and biotechnology companies: rare diseases being targeted, drugs acquired, and details of commercial alliances.
- Profiles of specialty rare-disease companies: platform technologies, therapies in development, and company financing and partnership agreements.
- Case studies of successful approaches for rare-disease drug development: high-throughput screening initiatives to uncover drugs that can exert therapeutic effects in rare diseases, Pfizer’s strategy of repurposing a current therapy for a highly lucrative rare disease, Novartis’s novel approach of studying disease pathologies to identify new targets and develop drugs with the potential to be used in multiple diseases.
- Indications mentioned: Muckle-Wells syndrome (MWS), cryopyrin-associated periodic syndromes (CAPS), familial cold autoinflammatory syndrome (FCAS), gout, gouty arthritis, juvenile idiopathic arthritis (JIA), type 1 diabetes, type 2 diabetes, impaired glucose tolerance (IGT), Duchenne’s muscular dystrophy (DMD), Becker’s muscular dystrophy (BMD), Hunter’s syndrome, Fabry’s disease, Gaucher’s disease, adenosine deaminase severe combined immune deficiency (ADA-SCID), metachromatic leukodystrophy, Wiskott-Aldrich syndrome, Pompe disease, hereditary inclusion body myopathy (HIBM), beta-thalassemia, sickle cell disease (SCD), childhood cerebral adrenoleukodystrophy (CCALD), lysosomal acid lipase (LAL) deficiency, HIV-1 infection, Wolman disease, nonalcoholic fatty liver disease (NAFLD), nonalcoholic steatohepatitis (NASH), mucopolysaccharidoses (MPS), gangliosidoses, Niemann-Pick type C (NPC) disease, pulmonary arterial hypertension (PAH).
Executive Summary
Strategic Considerations
Stakeholder Implications
Introduction
Corporate Strategies for Driving Drug Development for Rare Diseases
Novartis
GlaxoSmithKline
Pfizer
Focus on Rare-Disease Specialty Companies
Amicus Therapeutics
Ultragenyx Pharmaceutical
Bluebird Bio
Synageva BioPharma
Zacharon Pharmaceuticals
Development Through Public-Private Partnerships
Strategic Advantage of Drug Repurposing
NIH and FDA Initiatives
A Notable Example: Pfizer’s Sildenafil
Outlook for Rare-Disease Drug Development
Appendix A: Bibliography
About the Author
- AesRx
- Amicus Therapeutics
- Arch Venture Partners
- Avalon Ventures
- Bluebird Bio
- Cardiac Safety Research Consortium
- FDA’s Critical Path Initiative
- Duke University’s Clinical Research Institute
- FoldRx Pharmaceuticals
- Fondazione San Raffaele
- Fondazione Telethon
- Genethon
- Genzyme
- Genzyme Ventures
- GlaxoSmithKline
- Isis Pharmaceuticals
- JCR Pharmaceuticals
- Mayo Clinic
- National Center for Advancing Translational Sciences (NCATS)
- National Human Genome Research Institute
- National Institutes of Health (NIH)
- National Niemann-Pick Disease Foundation
- New Zealand Pharmaceuticals
- NIH’s Chemical Genomics Center
- NIH’s Therapeutics for Rare and Neglected Diseases program
- Nobelpharma
- Novartis
- Novartis Institutes for BioMedical Research
- Pfizer
- Prosensa
- Protalix Biotherapeutics
- Sanofi
- Shire
- Synageva BioPharma
- Third Rock Ventures
- Ultragenyx Pharmaceutical
- Zacharon Pharmaceuticals
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