Orphan drug designation is granted to drugs or biologics intending to treat, diagnose, or prevent rare diseases or conditions. Within the United States, a rare condition is one affecting under…
According to a recent article, Revolo Biotherapeutics announced it has obtained approval from the Medicines and Healthcare products Regulatory Agency (MHRA) for two Phase 2 clinical trials to analyze its…
In a February 8, 2022 news release, late-stage clinical company Synthetic Biologics, Inc. shared that the FDA granted Orphan Drug designation to the therapeutic candidate VCN-01. VCN-01 is a therapeutic…
People with epidermolysis bullosa (EB) often experience symptoms such as intense itching or severe pain. Unfortunately, these symptoms can be debilitating and may greatly affect overall quality-of-life. However, some burgeoning…
According to a recent article, the European Commission (EC) has approved Merck’s KEYTRUDA as an adjuvant treatment for type renal cell carcinoma (RCC), a type of kidney cancer. Renal Cell…
KalVista Pharmaceuticals plans to initiate a Phase 3 trial for KVD900, a treatment for hereditary angioedema (HAE) after finding positive results from earlier Phase 1 and 2 trials. The Phase…
It is common practice to treat multiple myeloma patients with a stem cell transplant. In the GENESIS trial, the stem cells were autologous (from the patient’s own peripheral blood or bone…
In a late January 2022 news release, gene therapy company Taysha Gene Therapies, Inc. ("Taysha") shared that trial data was now available on TSHA-120. The therapy, designed to treat giant…
Orphan Drug designation is granted to drugs or biologics intending to treat, prevent, or diagnose rare diseases or conditions. For the purpose of the designation, “rare” is considered any condition…
Cannabidiol (CBD) has recently been lauded for its many uses, ranging from stress relief to seizure reductions. Specialty pharmaceuticals company Zynerba Pharmaceuticals (“Zynerba”) developed an experimental cannabidiol gel called Zygel…
In a news release from January 24, 2022, immuno-pharma company AIM ImmunoTech Inc. ("AIM") shared that positive clinical data was now published. The data, which you can find in Clinical Cancer…
In late December 2021, biopharmaceutical company Deciphera Pharmaceuticals, Inc. ("Deciphera") shared that its therapy QINLOCK (ripretinib) was approved in the United Kingdom. The treatment is designed for adults with advanced…
According to a recent article, a new stem-cell-based gene therapy is showing promising results for treating beta (β)-thalassemia in a phase 1 clinical trial. Beta Thalassemia Beta thalassemia is a…
What if researchers could change DNA processes within brain tumors to improve overall survival rates and patient outcomes? According to Medical XPress, researchers found that L-methylfolate, in conjunction with the…
Clinical trial results can help support drug developers' goals. For example, positive trial results may support Marketing Authorization Applications (MAAs) or other areas needed to get therapies into the…
Back in December, UCB released the results of its phase 3 trial of rozanolixizumab in adult patients with generalized myasthenia gravis (gMG). Fortunately, these data were positive, pointing to this…
Over the last 10+ years, there have been extreme advances in the treatment of metastatic melanoma. In particular, researchers have sought to develop therapies which halted cancer progression and reduced…
Just 50 years ago, receiving a diagnosis of severe aplastic anemia could be devastating. While therapeutic options have greatly improved over the past five decades, some doctors have still found…
In a December 13 press release listed on the company's website, genome editing company Intellia Therapeutics, Inc. ("Intellia") shared that the first patient had been dosed in a Phase…
In a news release from mid-December 2021, rare liver disease company Albireo Pharma, Inc. ("Albireo") shared that positive topline data was available from a Phase 1 clinical trial evaluating A3907…
According to a December 16, 2021 news release from biopharmaceutical company Applied Therapeutics, Inc., the company has initiated a registrational Phase 2/3 INSPIRE clinical trial to evaluate AT-007 for patients…
According to a news release from RNA therapeutics company ProQR Therapeutics N.V. ("ProQR"), the first patients have been dosed in two Phase 2/3 clinical trials evaluating QR-421a for USH2a-mediated retinitis…
According to a recent article from Pharmacy Times, Octapharma’s cutaquig has been approved by the FDA to treat patients who are 2 years of age or older with primary humoral…
In 1960, the FDA approved chlorthalidone for hypertension (high blood pressure). However, doctors were unsure whether this therapy could benefit patients with advanced chronic kidney disease (CKD) who had high…
According to an article from the National Hemophilia Foundation, CSL Behring and uniQure have recently provided an update on their phase III HOPE-B trial. This study is evaluating etranacogene dezaparvovec,…
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