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Medgenics (LSE:MEDG) shares shot up 70.00 pence to 475.00, an increase of over 17%, this morning on the LSE. The share price has risen over 170% in the last twelve months as the company continues impressive development of its proprietary Biopump™ technology. Today’s investor reaction follows the announcement of a major step forward in the development of the company’s INFRADURE Biopump™.
FDA Approval Announced
Medgenics announced yesterday at 4:00 p.m. UCT that the US Food & Drug Administration had given the company’s INFRADURE Biopump™, a unique protein delivery treatment for patients with hepatitis D, an Orphan Drug Designation. This is the first Biopump™ based treatment to receive Orphan Drug Designation.
What is the INFRADURE Biopump™?
INFRADURE is a long-acting protein therapy that is capable of delivery a sustained dosage of interferon-alpha (IFN-α) for the treatment of hepatitis. The Biopump™ is a proprietary protein delivery system developed by Medgenics. A sliver of a patient’s skin is harvested and processed. It is then implanted back into the same patient where it will deliver the medication for six months. The current delivery method is typically by frequent, periodic injection, but is accompanied by temporary, but severe, side effects and is efficacious in no more than 50% of treated patients. An oral dose is also available, but has proven to have very little effect.
Medgenics’s plan is to use the Biopump™ platform to treat other diseases including anemia, hemophilia, multiple sclerosis, arthritis, diabetes, and diseases.
What is Orphan Drug Designation?
An orphan drug is typically one that has been developed for treatment of a designated orphan disease. An orphan disease is one that affects a relatively small percentage of the population, one which, therefore, pharmaceutical companies are less inclined to develop, as they would not expect to be able to recuperate the costs involved in development of a treatment. Perhaps the most renowned orphan disease is cystic fibrosis.
The purpose of the Orphan Drug Diagnosis is to provide the framework and incentives for sustained development of a designated drug. Although an orphan drug must conform to the same regulatory development path as any other drug, some burdens and constraints of the process are typically eased to create a quicker road to approval. Particularly popular in the US and the EU, the designation may also carry some other benefits in the form of research subsidies, tax incentives, and enhance patent and marketing rights. The designation is a means of cutting through red tape to get a product to market more quickly.
Expert Comments & Observations
Andrew Pearlman, President and CEO of Medgenics, said “We see this Orphan Drug Designation from the FDA as another important step in the regulatory pathway for our Biopump™ platform. The FDA’s timely approval of our Orphan Drug Designation for INFRADURE to treat hepatitis D is a key milestone in our broader hepatitis program as data gathered through clinical trials for INFRADURE in hepatitis D may serve as relevant support for other clinical uses of INFRADURE including hepatitis C and hepatitis B.”
Dr. Bruce Bacon, past President of the American Association for the Study of Liver Disease, observed that “INFRADURE offers the potential for a superior treatment for the 15 million people suffering from hepatitis D worldwide. The current treatment for hepatitis D requires years of weekly injections of interferon alpha, which leads to patient discomfort and substantial compliance challenges. Oral antiviral treatments have proven to be ineffective in treating hepatitis D. INFRADURE is intended to be implanted infrequently, with a single administration potentially replacing many months of weekly injections. This could offer a safe and efficacious treatment that could greatly improve patient compliance. The treatment also has potential for efficacy with greater patient compliance for other forms of hepatitis including the 170 million people infected with hepatitis C and the over 350 million people infected with hepatitis B.”
Dr. Marlene Haffner, former Director of Orphan Products Development at the FDA, added that “The novel Biopump™ platform potentially offers significant advance over current treatment not just for an orphan disease, but also for other diseases where protein therapy is a potential treatment.”
Company Spotlight
Medgenics is developing a pipeline of products based on its proprietary Biopump™ platform. Biopump™ is a “sustained action, therapeutic, protein delivery system” manufactured from a tiny sliver of the patient’s own skin. The Israeli company is targeting the protein therapy niche market which is project to represent some $132 billion per year by 2013.
