An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical...
An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease.
An orphan disease is often a rare disease which affects a small percentage of the population.
Without orphan disease designation, these rare diseases would probably be ignored by mainstream pharmaceutical and biotech development.
In the US and EU it is easier to gain marketing approval for an orphan drug, and there may be other financial incentives, such as extended exclusivity periods, all intended to encourage the development of drugs which might otherwise lack a sufficient profit motive.
The assignment of orphan status to a disease and to any drugs developed to treat it is a matter of public policy in many countries, and has resulted in medical breakthroughs that may not have otherwise been achieved due to the economics of drug research and development.
The intervention by government on behalf of orphan drug development can take a variety of forms:
- Tax incentives
- Enhanced patent protection and marketing rights
- Clinical research financial subsidization
- Creating a government-run enterprise to engage in research and development
Different countries manage orphan disease and drug designation in different ways, therefore it is important to establish if and how to obtain orphan designation prior to commencing on product R&D.
Due to the presence of orphan disease and drug designation, companies actively partner to develop and commercialize drug products for rare diseases.
Purchase the report Orphan Diseases Partnering Terms and Agreements
Recent deals with an orphan drug focus include:
Licensing agreement for Glybera
Xenon Pharmaceuticals, UniQure
Xenon has received a milestone payment from uniQure BV for the European Commission marketing approval of Glybera, a novel gene therapy treatment for the orphan disease lipoprotein lipase deficiency, and the first gene therapy approved in the Western world.
Xenon exclusively licensed to uniQure BV its rights to the LPLS447X genetic variant that causes a gain of function in the lipoprotein lipase (LPL) gene in humans.
In addition to the milestone payment Xenon is also entitled to receive from uniQure BV royalty payments on sales of Glybera, which is expected to start selling in Europe in 2013.
View the full deal record for this and other deals at CurrentAgreements, the life sciences partnering, M&A and financing deals database.
Or purchase the report Orphan Diseases Partnering Terms and Agreements
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