We are excited to share that Golden Heart Flower, Ltd. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the company’s product GHF-201 to treat glycogen storage disease type IV, including APBD.

Golden Heart Flower (GHF) – an Israel-based pharmaceutical company founded by a family with a member diagnosed with APBD – is focused on developing therapies for APBD and other rare glycogen storage and lysosomal storage diseases. GHF-201 is the company’s lead small molecule, discovered following a long study by researchers Dr. Or Kakhlon from Hadassah Ein Kerem Medical Center (Israel) and Professor Miguel Weil from Tel Aviv University (Israel). GHF-201 is capable of degrading polyglucosan bodies, the culprit in APBD. In an APBD mouse model study, GHF-201 was effective in lowering polyglucosan bodies, improving mobility, and extending lifespan.

In light of the unique mechanism of action of GHF-201 and encouraging results in laboratory studies and animal models, the company is also expanding its activities to include development of solutions for lysosomal storage diseases and other diseases with a higher prevalence in the overall population.

For over two years now, an “Urgent Compassionate Use Program” with GHF-201 has been running at the Hadassah Ein Kerem Medical Center (Israel) under the direction of Prof. Alexander Lossos and research coordinator Anat Mordechai. Israel’s Ministry of Health provided this approval to treat three APBD patients.

The FDA’s orphan drug designation supports the development of medicines for rare diseases or conditions that affect fewer than 200,000 people in the US. It gives the receiving pharmaceutical company significant incentives to promote the development of the drug. This includes the potential for seven years of market exclusivity once the drug gets marketing approval, eligibility for tax credits, fee waivers, and access to specialized regulatory advice from the FDA.

“We are excited to win the FDA’s recognition manifested in the granting of an orphan drug designation. This is an important milestone for the company in the development process of the GHF-201 drug that offers hope for people living with this serious disease. We would like to express our gratitude to our partners Hadassah Ein Kerem Medical Center, Tel Aviv University, and the Lyotropic Delivery Systems (LDS) Ltd., who supported the submission of the application for orphan drug designation. We are thankful for the APBD Research Foundation, the Israel-based patient organization MIFNEH, and our investors, without whose support, none of this would have been possible. We are currently raising funds to support the ongoing development of GHF-201,” shared Yaniv Glazer, CEO of GHF. Read the company’s statement…  

Jeff Levenson, DDS co-president of the APBD Research Foundation remarked, “Investing in Dr. Kakhlon’s research and development of GHF-201 over the years and seeing it reach this point, is a milestone for our community. Moreover, what we make possible for APBD patients has potential implications for patients with related disorders as well.”