Have you ever heard of a biosimilar? A biosimilar is essentially a biologic that is highly similar in structure and function to a reference product. Although this might seem technical, it really…
To the primary biliary cholangitis (PBC) community - it's time to rejoice! For the first time in nearly one decade, the U.S. Food and Drug administration (FDA) has approved a…
Ultomiris (ravulizumab) and Soliris (eculizumab), both developed by global biopharmaceutical leader AstraZeneca, are both effective treatments for paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). Yet this efficacy…
Pulmonary arterial hypertension (PAH), a rare and chronic disorder characterized by high blood pressure (hypertension) in the lungs that causes the pulmonary arteries to thicken or harden, most often manifests…
Pulmonary arterial hypertension (PAH) is a rare, chronic, and progressive disorder characterized by high blood pressure in the lungs. This causes pulmonary arteries to thicken or harden, which eventually puts…
The Duchenne muscular dystrophy (DMD) community has a reason to celebrate. According to a recent news release, the FDA recently approved Duvyzat (givinostat) for people ages six and older who…
In early March 2024, Tyler Patchen reported that the U.S. Food and Drug Administration (FDA) approved both intravenous and subcutaneous formulations of Tyenne (tocilizumab-aazg) for use in a variety of…
A majority of available treatment options for metachromatic leukodystrophy (MLD) rely on relieving symptoms. However, the recent approval of Lenmeldy (atidarsagene autotemcel) is the first ever FDA-approved gene therapy option…
On March 7, 2024, the United Kingdom’s Medicines and Healthcare products Regulatory Agency shared via press release that the agency has approved Ztalmy (ganaxolone) for people living with CDKL5 deficiency…
At the end of January 2024, global healthcare leader Eli Lilly and Company shared that its therapy OLUMIANT (baricitinib) received regulatory approval in Canada for the treatment of severe alopecia…
In January 2024, the FDA approved Hyqvia (Immune Globulin Infusion 10% [Human]) as a maintenance therapy for adults living with chronic inflammatory demyelinating polyneuropathy (CIDP), a rare neurological disorder. As…
People with chronic inflammatory demyelinating polyneuropathy (CIDP), a rare neurological disorder, now have another treatment option: Gammagard Liquid (immune globulin infusion [human] 10% solution). Healio reports that the Food and…
Immunotherapy, targeted therapy, surgery, radiation, chemotherapy, intravesical treatment - oh my! These therapeutic options have all been used to treat individuals living with urothelial carcinoma. But the treatment landscape has…
In 2015, the U.S. Food and Drug Administration (FDA) approved NUCALA (mepolizumab) for the treatment of severe eosinophilic asthma in adolescents and adults ages 12 and older, and by 2022,…
In the past, Hyqvia [immune globulin infusion 10% [human] with recombinant human hyaluronidase] was approved to treat people with primary immunodeficiencies (PIs) ages two and older. Now, following data from…
In 2015, the United States approved Kanuma (sebelipase alfa) for the treatment of infants living with rare lysosomal acid lipase deficiency. However, the therapy was not approved in other countries;…
In March 2022, the U.S. Food and Drug Administration (FDA) decided against the approval of oleogel-S10 for epidermolysis bullosa (EB); the FDA noted that, prior to approval, it would need…
In January 2023, the U.S. FDA approved Jaypirca (pirtobrutinib) for the treatment of adults with relapsed or refractory mantle cell lymphoma who had previously undergone two lines of systemic…
Also known as cholangiocarcinoma, biliary tract cancer can be incredibly aggressive. Further, this cancer is often diagnosed in later stages, making it more difficult to treat. The 5-year survival rate currently…
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired hematopoietic stem cell disorder in which blood cells break apart prematurely. In an effort to bring more effective and targeted treatments…
As shared by MedScape, the U.S. FDA recently approved the first treatment for people living with congenital thrombotic thrombocytopenic purpura (TTP). Its name? Adzynma. In the past, people with TTP…
In 2005, the U.S. Food and Drug Administration (FDA) approved abatacept for the treatment of moderate-to-severe rheumatoid arthritis. Twelve years later, in 2017, the FDA expanded the drug's approval…
In the past, the U.S. Food and Drug Administration (FDA) approved Opdivo (nivolumab) for different forms of cancer, such as non-small cell lung cancer (NSCLC) and melanoma. More recently,…
Previously, the FDA has approved Jardiance (empagliflozin) as a treatment option for adults with heart failure and to manage blood sugar in people ages 10+ with type 2 diabetes.…
Have you ever heard of CD55-deficient protein-losing enteropathy? This rare condition, which is also called CHAPLE disease, was only identified in the past 10 years. Right now, there are only…
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