First patient treated with Orpha Labs' drug

Orpha Labs AG has made use of the Compassionate Use Program for its drug ORL-101. This means that the drug, which has not yet received official authorization, can be used to treat patients suffering from the rare form of immunodeficiency LAD-II in cases where authorized therapeutics do not deliver the desired results.  

As Orpha Labs has now announced, the Israeli Ministry of Health has approved the use of ORL-101 within the framework of the Compassionate Use Program of Orpha Labs. The Zug-based BioTech firm, which specializes in discovering and developing treatments for ultra-rare diseases, writes in the press release that the first patient in Israel has now been treated with the drug.

“We are really pleased to announce this progress”, explains Alp Bugra Basat, founder and CEO of Orpha Labs, in the press release, before adding: “ORL-101 is the first product approved using a Compassionate Use Program for LAD-II patients. We have therefore taken one step closer to our ambition of bringing products to clinical development stage for patients suffering from extremely rare diseases”.

Orpha Labs intends to launch a Phase III clinical trial in the near future, the press release explains further. In the meantime, the United States Food and Drug Administration (FDA) may opt to review a marketing application for ORL-101 as a matter of priority.