Israeli treatment for congenital emphysema granted orphan status by FDA

The Kamada lab – we have an excellent safety profile with two viral elimination methods.Most people may not be familiar with its scientific name, but Alpha-1 Antitrypsin is one of the most common serious hereditary disorders in the world and …

The Kamada lab – we have an excellent safety profile with two viral elimination methods.Most people may not be familiar with its scientific name, but Alpha-1 Antitrypsin is one of the most common serious hereditary disorders in the world and can result in life-threatening liver disease in children and adults or in lung disease in adults.

Alpha-1 has been identified in virtually all populations. An estimated 100,000 Americans and a similar number in Europe have the severe deficiency – caused by the absence of a protein called an API – Alpha 1 proteinase inhibitor – which is produced in the liver.

An estimated 25 million people in the U.S. carry a single deficient gene that causes Alpha-1 and may pass the gene on to their children. You may know Alpha-1 by the more well-known name – congenital emphysema. The usually fatal disorder is a pulmonary disease in which lung tissue is damaged by the lack of API.

But now another hope for a long-term treatment for Alpha-1 is on the horizon thanks to Kamada – an Israeli biopharmaceutical company specializing in developing proteins from human plasma.

The Rehovot-based company was recently granted orphan drug status by the Food and Drug Administration (FDA) and the European Medicines Agency (EMEA) for the aerosolized version of its in-development API product. Simultaneously, Kamada has also developed an API product to be given intravenously, which recently completed registration in an unnamed country, and will begin marketing during 2005.

The FDA’s orphan drug designation is intended to encourage research and development of new therapies for diseases that affect fewer than 200,000 US residents. As a designated orphan drug, Kamada will receive assistance from the FDA in guiding the drug through the regulatory approval process. The designation provides the opportunity for Kamada’s API product to obtain market exclusivity for seven years from the drug’s approval date.

The company, which has 100 employees and scientists – develops, manufactures, and markets over 15 drugs in Israel and around the world.

“Basically, we produce products destined to save human lives,” Kamada Intellectual Property manager Pnina Strauss told ISRAEL21c. “The company which was founded 14 years ago, has its products distributed in more than 20 countries – most for congenital disorders or infectious diseases, as well as products not produced from plasma that are used in emergency situations like trauma and burn treatment.”

According to Strauss, congenital emphysema is an especially dangerous ailment because it usually sits dormant for many years.

“Usually symptoms begin to arise when the person in his 20s or 30s, and gradually the functions of the lung deteriorate. If you smoke or are exposed to highly polluted areas, it can aggravate the process,” she said, adding that only about 10% of those with congenital emphysema have been identified.”

According to the Alpha 1 Foundation, even though a simple blood test can disclose whether someone has the disease, it often takes an average of three doctors and seven years from the time symptoms first appear before proper diagnosis is made.

Kamada is competing with three international companies who also produce medicine to treat congenital emphysema, but Strauss says that not only is there room for all possible treatments for the disease, but Kamada’s has a number of advantages over the competition.

“There’s a considerable shortage worldwide resulting from limited availability of raw materials and facility production capacity. The patient need is not covered, and the yearly diagnosis and patient number is increasing and will continue to grow,” she said.

“Our intravenous API product is the only, liquid presentation “ready to use” product. This feature alone is more patient friendly. Additionally, our product is free of added substances – no sucrose or stabilizers, and we have an excellent safety profile with two viral elimination methods.”

The intravenous API is administered either by a nurse at home or many people go to clinics to get the product. It will be fully reimbursed by insurance in the US – and costs between $60,000-80,000 per person per year.

“The intravenous API is currently about to start Phase III trials and recently completed registration in an unnamed country,” said Strauss.

But the real feather in Kamada’s cap is their second generation API product which will be an aerosolized version. Kamada has invested over $25 million to date in developing the drug and expects the development of the inhalation version of the drug to take three years to complete.

The product – the first of its kind – is designed to be taken by inhalation to treat congenital emphysema, and according to Strauss, will be similar in form to an inhaler device like those used by asthma patients. However, she said, this is very preliminary and we will know more as we progress”.

“It won’t exactly be a cure – but a replacement therapy for the lack of the protein. Whether a person will fully recover depends at what stage of the disease you catch it. With age lung function ? even in a normal person – begins to decrease . With emphysema, it’s aggravated. The results of taking API is that the deterioration is not so severe, more like that of a normal person,” said Strauss.

Until Kamada’s treatments are commonplace in American clinics, The World Health Organization (WHO) recommends that all individuals with Chronic Obstructive Pulmonary Disease (COPD), as well as adults and adolescents with asthma (an estimated 30 million Americans) be tested for Alpha-1.