Rare diseases, or orphan diseases, affect a small percentage of the population. In the European Union, a disease is considered rare when it affects fewer than 1 in 2,000 people. In the United States, a disease is classified as rare if it affects fewer than 200,000 people nationwide. Despite their low prevalence, over 7,000 rare diseases collectively affect millions worldwide. These diseases are often chronic, progressive, debilitating, and life-threatening.
Challenges
The development of treatments for rare diseases presents unique challenges. Due to the small patient populations, there is often limited knowledge about the disease’s natural history, making it challenging to design clinical trials. Furthermore, the financial incentive for pharmaceutical companies to develop treatments for rare diseases is lower compared to more common conditions, as the market for such drugs is small. This has led to the designation of these conditions as “orphan” diseases because they were often neglected in research and development.
Regulatory Incentives
Regulatory agencies such as the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have established orphan drug programs to address these challenges. These programs provide incentives to encourage the development of treatments for rare diseases. Incentives include tax credits for clinical research costs, assistance with clinical trial design, waiver of regulatory fees, and market exclusivity for a period following approval.
Björklund Pharma’s Commitment
Björklund Pharma is dedicated to addressing the unmet medical needs of patients with rare diseases. The focus is on innovative research and the development of biopharmaceutical solutions that can significantly impact patients’ lives. Active exploration of the therapeutic potential of various compounds, including peptides, enzymes, and other biofactors that play crucial roles in physiological functions, is ongoing.
Thymosin Beta 4 in Multiple Sclerosis
One promising product candidate, thymosin beta-4 (Tβ4), is being investigated for its potential to treat multiple sclerosis (MS), which, due to its specific subsets and progression stages, can be considered a rare disease. Tβ4 has shown potential in promoting tissue repair and regeneration in various neurodegenerative conditions. This aligns with Björklund Pharma’s mission to pioneer treatments beyond symptomatic relief and targeting the underlying causes of diseases. Read More
Expanding Research in Rare Diseases
In addition to MS, Björklund Pharma is actively exploring other rare diseases where innovative therapies could make a significant difference. The company is investigating the therapeutic potential of natural compounds and bioactive ingredients that may offer new avenues for treating rare conditions. By leveraging cutting-edge science and innovative therapeutic approaches, Björklund Pharma aims to bring hope to patients and families affected by rare diseases.
Future Directions
Looking ahead, Björklund Pharma is committed to expanding its research portfolio to include more orphan diseases. The company strives to bring hope to patients and families affected by rare diseases by leveraging cutting-edge science and innovative therapeutic approaches. The commitment extends to collaborating with regulatory bodies, patient advocacy groups, and the broader scientific community to accelerate the development of effective treatments and ensure they reach those in need.
