Celebrating 40 Years of Orphan Drug Innovation
Orphan Drug development is central to what we at Xentria are all about. We are dedicated to pursuing ambitious drug innovation hoping to impact the lives of people living with rare diseases.
In 2023, we commemorate the 40th anniversary of the Orphan Drug Act in the US – a landmark legislation that has catalyzed remarkable breakthroughs in treatments for diseases that were once overlooked. These treatments now bring hope and relief to countless lives.
Prior to 1983 there were very few therapeutic options for those suffering from rare diseases. Here at Xentria, we are focused on developing treatment for these previously ‘orphaned’ diseases which pushes us towards greater levels of collaboration, tenacity and partnerships. Xentria places significant efforts into building authentic relationships with energy, vision and high levels of teamwork and trust.
Our lead candidate XTMAB-16 received Orphan designation in November 2020 and is currently under investigation as one of a few therapeutics specifically developed for the treatment of pulmonary sarcoidosis – a rare multisystem inflammatory disorder.
Cheryl Bradford, a patient living with the condition said: “Living with sarcoidosis is a journey filled with uncertainty, as this rare disease affects each person differently. It means facing physical and emotional challenges head on and finding the courage to persevere. It means embracing a life of adaptability, as symptoms can fluctuate, demanding constant adjustments. It means seeking support and connection, as the understanding of this condition can be overwhelming. But above all, living with sarcoidosis means finding hope and gratitude in the smallest victories, and never allowing the disease to define one's spirit.”
We continue development of XTMAB-16 with purpose and feel lucky to join a dedicated community of advocates and people working to make a change. Orphan Drug recognition highlights the persistent need for drug development in rare conditions and highlights the critical role of Orphan Drug Designation in the future of medicines for rare disease. To understand the impact of Orphan Drug Designation and the ongoing investments in treatments for unmet needs, we sought the perspective of those affected by sarcoidosis.
Paula Yette Polite, an executive director at the Sarcoidosis Research Institute and a patient herself, shared her story: “After a frustrating journey of very ill health, I was diagnosed with sarcoidosis. For more than 30 years I was treated with prednisone. For many years I felt isolated and alone but found solace communicating with other patients. Although patients have unique symptoms, deep bonds are established through conversations and shared experiences at group meetings and conferences. The Orphan Drug Act of 1983 has stimulated and incentivized the development of drugs for rare diseases. Forty years later, there are advancements of treatments for many rare diseases. Currently, there are more sponsored randomized clinical trials than ever before for the treatment of sarcoidosis.”
Tom Shea, President of Xentria said: “40 years on from the Orphan Drug Act in the US and the real need for ongoing drug development in rare conditions is still apparent. The act has enabled a true focus on developing treatments for conditions that were previously ignored or Orphaned. Gaining Orphan Drug Designation for our lead clinical candidate XTMAB-16 reinforced our commitment to pursue drug development specifically for patients living with this condition through a tailored drug development program.”
“The team remains dedicated to supporting those developing drugs with challenging profiles to take them through clinical development and one step closer to commercial launch.”
Sarcoidosis is a chronic, multisystem inflammatory disorder of unknown etiology that is characterized by the formation of granulomas — clumps of inflammatory cells — in one or more organs in the body. Sarcoidosis affects people of all ages but is most common among people who are 20 to 39 years of age.
While sarcoidosis can occur in any organ, more than 90% of patients with sarcoidosis will have the lungs affected, which is called pulmonary sarcoidosis. Left undiagnosed or untreated, the condition of patients with pulmonary sarcoidosis could degenerate into a chronic, progressive disease. Chronic, unresolved lung inflammation may result in scarring (fibrosis) that permanently damages the lung tissue and can lead to lung failure and death. In this complicated cascade of pro-inflammatory cytokines, the enhanced expression of TNFα, a cytokine that plays a significant role in antigen-stimulated, cell-mediated immune responses, may promote the formation of harmful granulomas and fibrosis throughout the body in people with sarcoidosis.
XTMAB-16 is chimeric human-murine anti-TNFα monoclonal antibody being developed as a novel biologic product for the treatment of pulmonary sarcoidosis with or without extra pulmonary involvement. Based on its ability to block TNFα, XTMAB-16 may disrupt an inflammatory pathway and help slow granuloma formation. Through dedicated regulatory work and focus from Xentria’s team, XTMAB-16 was granted Orphan Drug Designation for the New Drug Entity in November 2020. A Phase 1 clinical trial in healthy volunteers was completed in 2022.No TNFα inhibitor is currently approved for the treatment of sarcoidosis. Extensive analyses have been conducted to demonstrate the physio-chemical properties and pharmacology of XTMAB-16 as a TNFα inhibitor. The clinical study of this effect remains ongoing.
Established in 2020, Xentria works across biopharmaceutics communities creating innovative and authentic collaborations and partnerships that advance challenging drug development. Xentria, derived from “centrality”, is dedicated to delivering customized approaches to ambitious drug innovation through meaningful patient engagement and effective partnerships. Headquartered in Chicago, Xentria is taking the lead to support surging life sciences initiatives for global audiences, while nurturing diversity, individualism, and sustainability.
To learn more about Xentria visit www.xentria.com.