Orphan Drug Designation Granted for Potential Duchenne Muscular Dystrophy Treatment

For years FibroGen has been investigating a drug called pamrevlumab. They believe it could be an effective treatment for Duchenne muscular dystrophy (DMD), pancreatic cancer, and idiopathic pulmonary fibrosis (IPF). What do these three conditions have in common? They all are characterized by an overactive connective tissue growth factor which leads to excessive scarring, and ultimately organ dysfunction. Pamrevlumab is an antibody which works to inhibit this growth factor.

Pamrevlumab had previously received FDA Fast Track Designation for IPF as well as locally advanced unresectable pancreatic cancer. Additionally, it had received Orphan Drug Designation for IPF and pancreatic cancer. Now, FibroGen has just announced that pamrevlumab has finally received Orphan Drug Designation for DMD as well.

Orphan Drug Designation provides rare disease researchers a variety of advantages to assist in their development of novel therapies. It’s a small way to help accelerate the process of research and get new treatments into the hands of patients faster.

About DMD

DMD is a rare, debilitating, and fatal neuromuscular disorder caused by a missing dystrophin protein. This deficiency causes inflammation, fibrosis, muscle weakness, and muscle loss. The most severe effects of the disease surround the cardiac and respiratory muscles. Most patients are bound to a wheelchair before 12 years of age. After this non-ambulatory status occurs, other symptoms occur rapidly. These include scoliosis, joint contractures, cardiomyopathy, and even respiratory failure.

The vast majority of DMD patients are non-ambulatory. However, most DMD drugs have been created for ambulatory patients. There is clearly an unmet need for non-ambulatory DMD patients. FibroGen’s pamrevlumab program is working to fill this need.

Research Updates

FibroGen is currently working on Phase 3 clinical trials for both pancreatic cancer and IPF. Additionally, they have an ongoing Phase 2 clinical trial for DMD. So far, in all trials, pamrevlumab has shown both tolerability and safety.

This study has enrolled 21 non-ambulatory patients. All of these participants have reached the 52 weeks mark on the pamrevlumab treatment. Researchers are evaluating a wide array of factors including tissue fibrosis, lung function, muscle function in the upper extremities, as well as cardiac function.

Stay tuned to hear further updates on these exciting developments! In the meantime you can read more about this investigation here.


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