Rare Disease Report

Maha Radhakrishnan, M.D., Explains the Unmet Need in Orphan Diseases Like CAgD

DECEMBER 11, 2017
Maha Radhakrishnan, M.D.
Maha Radhakrishnan, M.D., Senior Vice President of Medical for Bioverativ Therapeutics, is justifably excited about her company's development of TNT009, a first-in-class monoclonal antibody intended for the treatment of the autoimmune hemolytic condition cold agglutinin disease (CAgD).

At present, there are no approved therapies for the chronic disorder CAgD, and it has been commonly associated with severe anemia. CAgD is characterized by its symptoms that can cause a significant burden on patients; including frequent blood transfusions, crippling fatigue, and an increased risk of life-threatening thrombotic events such as pulmonary embolism and stroke.

Rare Disease Report caught up with Radhakrishnan at the 59th American Society of Hematology (ASH) Annual Meeting and Exposition, and in this video, she discusses the unmet need for treatments of this rare disease, and rare diseases in general.



Radhakrishnan: I think the unmet need in the field is huge. I think the rare disease space is gaining traction just because of the fact that patients have no approved therapies or very few approved therapies. The standard of care is very low, and it’s upon us and moves us to actually elevate that standard of care, bring forward advances that are really bridging the unmet medical need. The therapies that are used sometimes today, even for agglutinin disease, have been used off label because physicians have no other choice and we have to give some improvement for patients. I think this is where the regulators are coming from; they’re actually working with the companies trying to help the advance the costs, advance in science and for example, even our program in agglutinin disease which we call 009 has received breakthrough therapy designation from the FDA. Which will hopefully help us expedite the program along pretty quickly working with the regulators.

So, when we talk about agglutinin disease I just want to spend a few minutes talking about just the shear work of the unmet medical need for patients. There are really no approved therapies today, you know. These patients go through a pretty significant burden in terms of what they need to endure. They have chronic anemia, which obviously is fatigue, shortness of breath, going through significant phases of hemolytic crisis. Hemolysis is something that is undermined in the minds of pathology. In addition, there’s also a huge burden of thromboembolic events in these patients. The study we presented yesterday on the natural history of this disease by Dr. Catherine Broome talked to us about it in terms of how high the burden is in terms of 55% increased chance of getting thromboembolic events. Physicians nowadays are using therapies like metuximab to give some benefit but these are unapproved. They look at other approaches of transfusions which again has its own side effects of iron overload and frequent need for transfusions. So, we’re already looking to bridge this gap by bringing forward a therapy which is our program which is a humanized monoclonal antibody looking to target the C1S which hopefully will help inhibit the antibodies that give a lot to the body that destruct the red blood cells. So, hopefully, we are hoping to tackle the anemia, hoping to tackle the hemolysis, hoping to reduce the burden thromboembolic side effects and give patients back the meaningful quality of life.

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