FDA Grants TauTona Group Orphan Drug Designation for Treatment of Sickle Cell Disease Ulcers

There is no standard of care for sickle cell patients who suffer from these debilitating wounds

TauTona Group, a vertically-integrated operational venture fund focused on creating solutions for unmet clinical applications in healthcare, announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to one of the fund’s investments for the treatment of skin ulcers in patients with sickle cell disease. Theris Medical, a TauTona Group company, will be commercializing the product.

Sickle cell disease is a genetic red blood cell disorder that affects hemoglobin, the molecule in red blood cells that transports oxygen through the bloodstream. People with this disorder have atypical hemoglobin molecules called hemoglobin S, which can distort red blood cells into a sickle, or crescent, shape. The disease affects approximately 100,000 patients in the US alone.

Of these sickle cell patients, approximately 14,000 are affected by leg ulcers, persistent wounds of the lower legs and ankles, which are extremely painful, difficult to treat, and frequently recurrent. Sickle cell ulcers can take many months to heal and nearly 30 percent relapse within 6-8 months. Because these wounds are known to be incredibly painful, painkillers such as opioids are often prescribed to obtain some relief. These ulcers severely impact the quality of life including ankle mobility, gait, sleep, self-esteem, and social and professional interactions.

“We are excited to receive an orphan designation for treating sickle cell leg ulcers, an extremely painful condition which has no FDA-approved treatments on the market today. At TauTona, one of our critical missions is to solve clinical needs in wound care, and this orphan designation in the US confirms the high unmet medical need for a very underserved population,” said Dr. Geoff Gurtner, co-founder and managing director of TauTona Group.

This is the first biopharmaceutical innovation created by the team at TauTona Group and based upon technology developed over years of research in Dr. Gurtner’s lab at Stanford University. In his research, Dr. Gurtner identified that deferoxamine, a drug sickle cell patients already use intravenously for removing excess iron in the blood could have a favorable effect on healing sickle cell leg ulcers if applied topically. TauTona licensed the technology to transition the novel wound care treatment from the lab to the clinic.

The Orphan Drug Designation program provides orphan status to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases and disorders that affect fewer than 200,000 people in the US, or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.
About TauTona Group
TauTona Group is a vertically-integrated operational venture fund that ideates, invests and develops new solutions in medtech, biotech and regenerative medicine that address unmet clinical needs in healthcare. With a strong belief that improvements in healthcare and cost reductions can only be achieved through innovation, TauTona’s sustainable investment model enables disruptive ideas to be evaluated, designed, tested and developed in an extremely economical manner benefitting all stakeholders: inventors, investors, doctors, the healthcare system, and, most importantly, patients. The fund was founded in 2010 by two Stanford School of Medicine professors and researchers focused on bioengineering, tissue regeneration, and stem cell therapeutic approaches. For more information, visit www.tautonagroup.com and www.therismedical.com.

For the full release, click here.