Ten million people around the world need treatment annually for rabies, and over a billion people live in areas of the world where rabies — contracted from dogs, bats, rats, or other animals — is a daily danger.
Yet rabies is classified as an “orphan disease.” Not because it’s rare, but because there are few commercially available vaccines to treat it. In the US, for example, only one company, Novartis, markets rabies prophylaxis treatments for humans, and worldwide there are only a few other companies serving the market.
That is set to change next year as Israeli biotech firm Kamada said Wednesday that it planned to apply for a Biologics License Application (BLA) from the US Food and Drug Administration, the last step before offering a remedy for sale in the United States.
Kamada’s KamRAB product is ready for the market, after successful completion of phase 2 and 3 trials in which, the company said, the product proved to be at least as effective as other rabies immunoglobulin (IgG) products.
Already being sold in 10 countries around the world, including Israel, KamRAB — and a version it plans to market with Italian pharmaceutical firm Kedrion, called KedRAB — is produced from the blood of human donors who have been exposed to rabies, on the theory that exposure enhances the development of antibodies. In development for over a decade, Kamada has designed a system to pasteurize and cleanse the IgG, using approved FDA blood-cleansing products.
In the trial, Kamada administered the serum to patients, broken up into groups based on less or more advanced stages of the disease. A control group was given IgG products already in use for comparative purposes. The supervised study of 118 subjects, Kamada said, yielded positive results for KamRAB, and that it had “successfully met the trial’s primary endpoint of non-inferiority when measured against an IgG reference product,” meaning that it was at least as good as existing products.
Rabies is a viral disease that causes acute inflammation of the brain in humans. Individuals can become infected when either bitten or scratched or by being exposed to infected animals’ saliva through broken skin, or the eyes, nose or mouth. If it isn’t caught and treated early, rabies nearly always results in death, usually within three months; less for children, as the virus has less distance to travel before attacking the central nervous system.
It’s for that reason that doctors usually insist on administering rabies vaccine, especially to children, even if it’s not clear if the animal in question was rabid. However, with limited supplies, it’s first-come, first-served for rabies viruses to the extent that shortages sometimes develop.
Earlier this year, for example, Australia’s public health body said that it has decided to begin importing made-in-Israel KamRAB because the country had exhausted its supply, even though it is not registered for use in Australia and not officially tested.
Kamada is a public company that specializes in treatments for orphan diseases, such as cystic fibrosis, cytomegalovirus (CMV) infection (which often affects transplant patients), Varicella Zoster herpes, hemophilia and other conditions. All of them affect millions of people around the world, but for various reasons have not been deemed worthy of extensive drug development by the large pharmaceutical firms.
Amir London, Chief Executive Officer of Kamada, sees an important role for KamRAB in many countries facing the same supply issues.
“Since 2003 we have marketed KamRAB in 10 countries outside the US and we have sold more than one million vials to date, which provides significant experience with this product,” London said. “We are confident our product will provide a much-needed, high-quality source of IgG for people who are at risk of being exposed to rabies.”
“We believe that a competitive product in the US will provide diversity of supply in a market with ample room for a new product entry,” he added.