Release Date: 13-Feb-2023
Rare diseases have smaller patient bases when compared to other disease prevalent in the general population, which deters pharmaceutical companies to consider these diseases while developing new drugs. It has been estimated that 7000 rare diseases have been identified till date which are both hereditary or acquired and out of these, approximately 95 percent do not have an effective treatment approach. As a result, the need of patients suffering from different rare diseases is highly unmet. However, the recent years have seen an expansion in the pipelines for such diseases, which has given a new hope to patients suffering from these diseases.
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Over the years, several countries including the US, EU, Australia and Japan have passed acts and legislations to encourage more pharmaceutical companies to develop drugs for rare diseases, termed as orphan drugs. As a result of this, a number of prominent companies have accommodated rare diseases and orphan drugs in their pipelines. These include Sanofi, Novartis, Ultragenyx, Pfizer, Pierre Fabre and Alexion (now acquired by AstraZeneca). A number of drugs have now been approved for different rare diseases, while a huge number of them have been granted the orphan drug designation. The FDA reportedly granted the orphan drug designation to more than 400 drugs and active substances in the year 2022 and more than 700 drugs with the designation not necessarily granted in 2022, were granted market authorizations.
Recently on February 8, 2023, the US FDA granted the orphan drug designation to BEA-17, a LSD1 inhibitor developed by Sweden-based Beactica Therapeutics for the treatment of glioblastoma, a rare but highly aggressive form of brain cancer. LSD1 interacts with CREBBP and by blocking this interaction BEA-17 prevents the glioblastoma tumors from overcoming stress and maintaining the progression of the tumors. Therefore, drugs designed to bind to the LSD1 protein have emerged as a viable therapeutic approach for the treatment of glioblastoma.
Similar to this, Roche's Rituxan, which is marketed as rituximab, received the orphan drug designation in January 2023 to treat membranous nephropathy. The new granting of this designation has given another indication to Rituxan's portfolio, which will assist grow its sales. Rituxan was previously approved for the treatment of hematological cancers, as well as other inflammatory and autoimmune diseases. Membranous nephropathy, which is characterized by a decline in kidney function, is a rare disease affecting 8andndash;10 individuals per 1 million people worldwide.
Rare diseases are much less prevalent than other common illnesses in the general population. Patients frequently accept off-label medication, which may not be as effective, in order to control their symptoms. Therefore, drug regulatory bodies provide incentives to drug makers such as tax credits for qualified clinical trials, exemption from user fees, potential market exclusivity after approval, and reduced annual fees depending on the country and regulatory body to encourage the development of drugs for orphan diseases.
The pipeline for rare diseases has been consistently expanding as a result of actions taken by pharmaceutical companies like AstraZeneca, Novartis and Roche. The awareness of these diseases has been increasing the drug research and development sectors and novel drugs developed using the latest technologies have displayed promising results. Patients with these diseases now have hope because more sophisticated approaches to directly target the underlying molecular causes are being used to treat them. The pipeline is expected to increase even more in the foreseeable future as a result of the increasing patient base as well as the initiatives taken by various central governments and drug regulatory agencies to promote the development of drugs and therapies for various rare diseases.