Cases of multiple myeloma seem to be rising across the country and the globe. New and effective therapeutic interventions have the potential to significantly benefit this patient population. Ichnos Sciences…
It can be difficult to spur interest in rare disease drug development. Forty years ago, the Orphan Drug Act passed as a way to incentivize drug development within this space.…
In the United States, Orphan Drug designation is granted to drugs or biologics being developed to treat, prevent, or diagnose a rare disease. These are conditions affecting fewer than…
Initially, belimumab (sold under the brand name BENLYSTA) was approved for the treatment of patients for lupus and lupus nephritis. It was the first FDA-approved biologic treatment within this indication.…
In the United States, Orphan Drug designation is granted by the FDA to drugs focused on the treatment, prevention, or diagnosis of rare conditions; these are those affecting fewer than…
In the past, it was difficult to incentivize the development of treatments for rare diseases, given issues with both profitability and small patient populations. However, all people are deserving…
Over 30 million people in the European Union (EU) are living with a rare disease. Yet rare disease drug development is still somewhat stifled. Orphan designation helps to incentivize drug…
It has historically been difficult to encourage the development of therapies for patients with rare diseases. To overcome this, the Orphan Drug Act was passed in 1983. This act…
In the European Union (EU), Orphan designation, or Orphan Medicinal Product designation, is granted to drugs or biologics intending to treat, diagnose, or prevent a rare, chronically debilitating, or…
In the United States, the Food and Drug Administration (FDA) grants Orphan Drug designation to drugs or biologics which intend to treat, diagnose, or prevent rare conditions. “Rare” conditions are…
In early January 2023, Healio reported that IOA-244, a treatment designed for uveal melanoma, was granted Orphan Drug designation by the U.S. Food and Drug Administration (FDA). Orphan Drug…
Orphan Drug designation is granted by the FDA to drugs or biologics intended to treat, diagnose, or prevent a rare disease or condition. “Rare,” in the United States, is…
Rare diseases are, by their nature, rare—and because of this, it can be difficult to spur drug development. The FDA worked to overcome this through the creation of the…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. These are defined as conditions affecting fewer…
Mahesh Karande, the President and CEO of Omega Therapeutics, is incredibly passionate about epigenetics. And it shows: in the work that he and his team do, in the therapies they…
It can be extremely difficult to incentivize drug development for rare diseases or conditions. To combat this, decades ago, the FDA created the Orphan Drug Act. Now, Orphan Drug designation…
The Orphan Drug Act was passed in 1983 to stimulate the development of therapeutics for rare diseases. With this came the Orphan Drug designation. This designation is granted to drugs…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose a rare disease or condition. Rare conditions are those affecting fewer…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. A rare condition is one affecting fewer…
According to a September 2022 article from OncLive, the European Medicines Agency (EMA) granted Orphan Drug designation to CAN-2409. This therapy is being developed as a potential therapeutic option for…
In a late September 2022 news release from AVROBIO, Inc., the company’s gene therapy candidate AVR-RD-04 recently earned Rare Pediatric Disease designation. This investigational therapy is being developed as a…
Osteosarcoma is genomically complex, which means that – sometimes – it can also be difficult to treat. Treatment and management, however, are crucial to ensuring positive patient outcomes. Doctors and…
In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to prevent, diagnose, or treat rare conditions. These are conditions affecting fewer than 200,000 Americans.…
According to an August 2022 news release from biotechnology company Elixiron Immunotherapeutics (“Elixiron”), EI-1071, a therapy for patients with idiopathic pulmonary fibrosis (IPF), recently earned Orphan Drug designation from…
Have you ever heard of Orphan Drug designation? This designation is intended to spur research and development into drugs or biologics for rare diseases. Orphan Drug designation is granted…
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