The most common molecule type for rare disease active pipeline drugs is gene-modified cell therapies, in which cells are genetically modified outside the patient’s body in order to help fight a disease. This contrasts with the larger pipeline, which is dominated by monoclonal antibodies, found GlobalData, a leading data and analytics company.

Camila Dalitz, Analyst for Drugs Databases, comments: “This trend will increase the number of gene-modified cell therapies in the rare diseases market in comparison with the overall market in the future, highlighting the use of gene-modified cell therapies within niche areas such as rare oncology indications.”

According to GlobalData, there are over 10,000 drugs indicated for rare diseases under development, of which 13% are gene-modified cell therapies and 10% are monoclonal antibodies. In comparison, 11% of overall pipeline drugs are monoclonal antibodies and only 7% are gene-modified cell therapies.

According to the FDA and the NIH Genetic and Rare Diseases Information Center (GARD), rare diseases are indications that affect fewer than 200,000 people in the US. In the EU, the European Commission considers a condition a rare disease when no more than one person is affected in 2,000.

Dalitz concludes: “With recently approved drugs like Carvykti, we expect to see the number of gene-modified cell therapies on the market grow for rare diseases in the coming years. This shows the importance of gene-modified drugs in the treatment of rare diseases and how the number of gene-modified drugs is likely to grow in the future within niche markets overall.”