Investors fully believe in the new drug against a rare disease that the Leiden biotech company Pharming is marketing in the US. The stock rose by tens of percent. The drug costs $547,000 per patient per year.
It revolves around the drug Joenja, which is used against the rare hereditary disorder of the immune system APDS. In the Western world it is estimated that about 1500 people suffer from this disease. ,,APDS is a genetic defect of the immune system. As a result, the system produces only inactive immune cells. This causes a downward spiral from birth. Organs swell, the lungs become damaged and eventually the patients often develop lymphoma and death. Patients do not live very old due to all these disorders. A third will not be older than 60,” says Sijmen de Vries, CEO of Pharming.
Hefty price tag
With the new treatment, the patient can look forward to a normal life is the expectation. There is a hefty price tag, because the medicine has to be taken for life, two pills a day.
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Now we are no longer a one trick pony. Two drugs on the market is unique for a European biotech company
Analysts expect the new drug to bring in hundreds of millions in additional sales. Especially if Europe and Japan also allow the product on the market. There are an estimated thirty patients in the Netherlands who suffer from APDS. They may hear later this year whether the drug will be on the market.
Then the price may be one thing. De Vries does not yet know how expensive the medicine will be here. “But on average the prices for these types of orphan drugs in Europe are 60 to 70 percent of the price in the US.” De Vries points to an advantage. “Now patients often have to go to the hospital for treatment of the symptoms. Those high healthcare costs are avoided with the drug. And it improves the quality of life.”
One-trick pony
Until now, Pharming had one drug on the market, Ruconest, for hereditary angioedema. This generates about 200 million in turnover per year for the company. “Now we are no longer one one trick pony” De Vries notes with satisfaction. “Two drugs on the market is unique for a European biotech company.”
Joenja does not come from her own stable, but was bought from pharmaceutical giant Novartis. For that company, such a drug for a rare disease is actually difficult. They focus on drugs for common diseases that generate large sales. “We are good at commercializing medicines against rare diseases,” says De Vries. “There were nine companies that wanted this medicine. We have been chosen by Novartis.”
Attractive party
De Vries hopes to be able to grow considerably in the future with the purchase of these types of medicines. “We have transformed from a one-product, one-market company to a two-product, multi-market company. That makes us attractive as a party to market these types of medicines.”
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