Omeros has filed an orphan drug application with the US FDA for OMS721, used to treat atypical hemolytic uremic syndrome (aHUS). OMS721 works by blocking the lectin pathway of the complement system while leaving intact the classical ...
Tags: Omeros, Orphan Drug, Medicine
Neuralstem has obtained notice of allowance for a patent that covers various methods to use expanded spinal cord stem cells such as NSI-566 to treat amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). The company completed Phase ...
Activartis has received European orphan drug designation for a cancer immune therapy, AV0113, for use in the treatment of glioma. AV0113, which was developed against every type of cancer, is based on a procedure where the immune system ...
Tags: orphan drug, cancer immune therapy
The USFDA has granted orphan drug designation to Pluristem Therapeutics' PLacental eXpanded (PLX) cells to treat aplastic anemia. The failure of hematopoietic stem cells (HSCs) present in the bone marrow to produce red blood cells, white ...
Tags: orphan drug, aplastic anemia, drug
The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to SOV Therapeutics' oral testosterone undecanoate, a pro-drug of testosterone, for the treatment of Constitutional Delay of Growth and Puberty (CDGP) in ...
Tags: orphan drug, oral testosterone replacement therapy, FDA
The European Commission and the European Medicines Agency have issued orphan drug designation to PROLOR Biotech's hGH-CTP. The longer-acting version of human growth hormone, hGH-CTP, is being developed to treat growth hormone deficiency. ...
Tags: PROLOR Biotech, hGH-CTP, European Orphan Drug Designation
Clinical stage biopharmaceutical company Apogenix's Apocept (APG101) has won FDA orphan drug designation to treat myelodysplastic syndromes (MDS). Apogenix also announced the commencement of Apocept Phase I trial in MDS patients in ...
Tags: FDA, orphan drug, biopharmaceutical
The US FDA has granted Soligenix fast track designation for OrbeShield for the reduction of mortality associated with gastrointestinal acute radiation syndrome (GI ARS). The oral beclomethasone 17,21-dipropionate (oral BDP) earlier ...
Tags: US FDA, Soligenix, fast track designation, OrbeShield
Telik has announced notification of FDA orphan drug designation to ezatiostat HCL (Telintra) for the treatment of myelodysplastic syndrome (MDS). The novel inhibitor of the enzyme glutathione S-transferase P1 1,Telintra activates Jun ...
Tags: orphan drug, ezatiostat HCL, drug
PharmaIN and LAT Pharma have announced the issuance of FDA orphan-drug designation for terlipressin as treatment of ascites due to all etiologies apart from cancer. The companies are developing long-acting PHT101(PGC-C12E-Terlipressin)for ...
Tags: PharmaIN-LAT Pharma ascite therapy, FDA orphan-drug designation, PHT101
GeNO has received four additional US patents for its proprietary nitric oxide (NO) generation chemistry and delivery technology. The patent No. 8,211,368 describes the conversion of nitrogen dioxide (NO2) to NO where NO2 is stored as ...
Tags: US patent, clinical technology, clinical research, GeNO
The US FDA has granted orphan drug designation to Milo Biotechnology's AAV1-FS344 to treat Becker and Duchenne muscular dystrophy. AAV1-FS344 is myostatin inhibitor delivered through gene therapy to increase muscle strength in patients. ...
Tags: FDA, orphan drug designation, US
The FDA has granted orphan drug designation for Andromeda Biotech's DiaPep277 to treat type 1 diabetes (T1D) patients with residual beta cell function. The orphan drug status offers seven years marketing exclusivity from the time of ...
Tags: DiaPep277, type 1 diabetes, T1D, residual beta cell
CSL Behring has received FDA orphan drug designation for its recombinant fusion protein linking coagulation factor IX with recombinant albumin (rIX-FP). The designation was granted for the treatment and prophylaxis of bleeding episodes in ...
Tags: CSL Behring, rIX-FP, hemophilia B
Threshold Pharmaceutical has received orphan drug designation for TH-302, a cancer drug, from the US FDA. Threshold is presently testing TH-302 along with a commonly used chemotherapeutic agent, doxorubicin, in a late-stage trial to treat ...