Case Study

Creating A New Dosage Form From An Approved Drug For A New Rare Disease Indication

By Sundeep Sethia, Senior Director, Pharmaceutical R&D; Veeranna Lolla, RAC, Senior Director, Regulatory Affairs; Shafi Shaik, Principal Scientist, Product Development

science lab clinical trial

Armed with a promising idea and an urgency to help patients with Hereditary Hemorrhagic Telangiectasia, Cure HHT contacted Pii for assistance in developing a formulation and filing an Investigational New Drug (IND) application with FDA.

Cure HHT is a foundation representing a group of patients, and their families, suffering from a genetic blood vessel disorder that causes bleeding in multiple organs of the body. This disease affects males and females of all ages and from all ethnic and racial backgrounds. The genetic mutation causes blood vessels to form abnormally and lack normal capillaries between arteries and veins. Patients with this disorder experience arterial blood under high pressure flowing directly into low-pressure veins without first having to move through small capillaries. The area where the artery connects directly to the vein tends to be a fragile site that can rupture and bleed, and tissue in the areas receive a decreased amount of oxygen as well. Symptoms can appear as re-occurring nosebleeds or as serious strokes and heart attacks. There is currently no treatment for this disease, and therapies focus on treating the symptoms only.

A pharmaceutical company, developing a molecule for another indication, discovered side effects during clinical trials that might mitigate or even cure the rare hereditary blood disorder. The initial clinical work began for the rare blood disorder, but due to a variety of unexpected circumstances, the program was never completed. However, a clinician that had worked on the original project began working with Cure HHT to reestablish the project.

Led by a passionate clinician who had experience with the molecule, Cure HHT believed they had the ability to pursue a cure. The project was re-started, an Investigational New Drug (IND) application was filed, but the FDA rejected it, placing the project on a clinical hold, because it lacked adequate testing and controls. The foundation was disappointed by the set-back but remained stalwart in their intent to find a solution.

Cure HHT possessed a wealth of information on the rare disease and direct clinical experience yet lacked a feasible formulation with adequate testing and controls. Pii was confident they could help.

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