Orphan drug designation was first introduced to the European Union in 2000. The designation is granted to therapies intended to treat, diagnose, or prevent a rare, life-threatening, or chronically…
Have you ever heard of Orphan Drug designation? This special status was created to incentivize the development of therapies for people with rare conditions (affecting fewer than 200,000 people…
Earlier this year, the FDA granted Accelerated Approval to ELEVIDYS (delandistrogene moxeparvovec-rokl) for the treatmnt of Duchenne muscular dystrophy (DMD) in boys aged 4-5. While this is a stunning…
According to an article from Akosua Mireku of Pharmaceutical Technology, the U.S. Food and Drug Administration recently granted Orphan Drug designation to SNB-101, a potential therapy for people living with…
The Orphan Drug Act of 1983 was implemented after the U.S. Food and Drug Administration observed drug development issues in the rare disease space. Rare diseases often have smaller…
Orphan Drug designation is granted to therapies that are designed to treat, diagnose, or prevent rare diseases or conditions. In the United States, a rare condition is one affecting fewer…
The Orphan Drug Act of 1983 transformed the rare disease treatment landscape. Prior to the Act’s passage, there was little commercial investment in drug development targeted towards rare conditions.…
Medical research has yielded a significant number of breakthroughs that have advanced the field and helped people in need. But in the rare disease sphere, it has sometimes been…
Throughout the world, it can be difficult to advance research and drug development in the rare disease space. In part, this is because many rare diseases have small population…
In May 2023, Pharmaceutical Technology reported that INO-3107, a DNA medicine vaccine candidate developed by biotechnology company Inovio Pharmaceuticals (“Inovio”), received Orphan Drug designation from the European Commission. INO-3107…
Mesothelioma can sometimes be difficult to treat. The cancer can spread to nearby tissue and nerves, and often isn’t diagnosed until later stages. Admittedly, there are a number of…
Earlier this year, I interviewed Dr. Arthur Suckow, the CEO and co-founder of DTx Pharma, about the innovative RNA therapeutics being developed for Charcot-Marie-Tooth disease type 1A (CMT1A). In…
Although the 5-year survival rate for pancreatic cancer has slightly risen (from 11% to 12%), there is still much work to be done. Pancreatic cancer can be difficult to treat.…
810 words (source) vs. 506 words (mine) - 2% match Chemotherapy, radiation, and surgery may all be utilized to help patients to manage nasopharyngeal carcinoma (NPC), a rare and aggressive…
As reported in Yahoo! Finance, the United States’ Food and Drug Administration (FDA) recently granted Orphan Drug designation to MP1032. This therapy, developed by clinical-stage biotech company MetrioPharm, is being…
Have you ever heard of the Orphan Drug designation? This designation, created through the Orphan Drug Act and granted by the U.S. Food and Drug Administration (FDA), is used to…
On May 18, 2023, Market Watch reported that rucosopasem manganese received Orphan Drug designation. Developed by clinical stage biopharmaceutical company Galera Therapeutics (“Galera”), rucosopasem manganese is largely being developed…
Pyxis Oncology is driven to improve the lives of people living with cancer through the development of innovative immunotherapy and antibody drug conjugate (ADJ) therapies. Their treatment PYX-201, a novel…
A therapy is granted Orphan Drug designation if the FDA believes that this therapy will treat, prevent, or diagnose rare conditions. Rare conditions, in the United States, are those affecting…
It’s no secret that, in many cases, funding for rare disease research and drug development is slim; the financial burden often falls to families and patients. 40 years ago, the…
In February 2023, I spoke with Dr. Allen Davidoff of XORTX Therapeutics Inc. (“XORTX”) about the company’s commitment to developing therapies for people living with progressive kidney disease. We…
The Orphan Drug Act of 1983 was designed to facilitate the development of orphan drugs, or drugs intended for rare or life-threatening indications. In the United States, rare conditions are…
In the United States, Orphan Drug designation is granted by the U.S. Food and Drug Administration (FDA) to drugs or biologics that are being developed for rare conditions. Rare…
Drug development within the rare disease space can be tricky. In many cases, there is a general lack of research and understanding around these conditions; as a result, many…
In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to diagnose, prevent, or treat rare conditions (those affecting fewer than 200,000 people in…
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