White paper: Development and production of orphan drugs. Tailored solutions by Lab Ofichem

We are proud to present our latest whitepaper on the importance, challenges, and opportunities of orphan drug development and the important role of CDMOs.

This whitepaper explains the significance and challenges of orphan drug development and the important role of Contract Development and Manufacturing Organizations (CDMOs). It describes how Lab Ofichem provides tailored solutions to accelerate orphan drug substance development and successful market entry.

Orphan drugs and rare diseases

Orphan drugs are designed to treat rare diseases. In the EU, a disease is defined as rare when it is affecting fewer than 1 in 2000 people in the community. According to the FDA, a rare disease is defined as a disease or condition that affects fewer than 200 000 people in the United States, which is of a similar order of magnitude.

Challenges in development

There are several challenges when it comes to developing orphan drugs. Most importantly, developing a new chemical entity is very expensive, and the limited market for orphan drugs makes it difficult to recoup these high development costs. For many companies, the development of novel orphan drugs is not economically interesting. The development of a repurposed drug could help reducing the costs, but efficient resource allocation with phase-appropriate support remains crucial.

Besides addressing the direct unmet medical need, another benefit of the improved research on rare diseases is the high amount of innovation: developing orphan drugs can drive innovation of pharmaceutical development, leading to new insights that may benefit broader patient populations.

The role of Lab Ofichem in the orphan drug landscape

Lab Ofichem is a European CDMO specialized in the development and subsequent GMP manufacture of small molecule niche APIs. With nearly 50 years of experience in the development and production of a broad variety of APIs, the family-owned company has a strong reputation for reliability and quality. Because the company is specialized in niche APIs, including multiple repurposed APIs with an Orphan Drug Designation, the team has a solid experience in the development and manufacturing of orphan drugs for both clinical batches and commercial production, and has a long history of regulatory compliance.