TXA127: Tarix Orphan LLC’s lead compound TXA127 has been granted Orphan drug status. It is a potential treatment for epidermolysis bullosa (EB), a connective tissue disorder. TXA127 impedes the work of the transforming growth factor (TGF) beta pathway which appears to play an important part in the development of EB.

SB 525: Sangamo Therapeutics and Pfizer have announced their product SB-525 has been granted orphan drug status by the EMA. This is a gene therapy product for the treatment of haemophilia A. Sangamo have also announced they are launching a phase I/ II clinical trial to evaluate the safety and tolerability of SB-525 in adults with severe haemophilia. Early data from this study is expected in late 2017 or early 2018. SB-525 reduces or eliminates the need for coagulation factor VIII replacement therapy by getting cells to naturally produce the missing protein. It works by carrying and delivering genetic information to liver cells where coagulation factors are produced using a modified, harmless form of the adeno-associated virus.

E4: Belgium based drug maker Mithra Pharmaceuticals’ estretol (E4) has received orphan drug designation for the treatment of neonatal encephalopathy (NE). The drug is intended to treat a specific type of NE known as hypoxic ischemic encephalopathy (HIE) which accounts for up to 80% of cases, affecting around 30,000 neonates per year in the EU and USA. The drug maker also plans to expand the use of E4 in other indications such as neuroprotection and wound healing.

Actimab-A: Actinium Pharmaceuticals have announced their drug Actimab-A has been granted orphan drug designation in the EU. It is intended for the treatment of newly diagnosed acute myeloid leukaemia (AML) in patients over the age of 60 that do not qualify for standard induction therapy. Actimab-A is currently in a multicenter phase II clinical trial that will enrol 53 patients. It targets the protein CD33, which is expressed on the surface of AML cells via the monoclonal antibody HuM195 which carries the potent cytotoxic radioisotope actinium-225 to the AML cancer cells. Actinium-225 emits high-energy alpha particles as it decays which destroys cancer cells. As the actinium-225 decays, it produces a series of daughter atoms, each of which also emit their own alpha particle which in turn, destroys more cancer cells.

Avacopan: Drug maker ChemoCentryx have been given orphan drug designation for their drug avacopan (CCS168) indicated to treat the kidney disease C3 glomerulopathy (C3G). This disease is characterised by deposits of proteins from the body’s complement system into and around the glomeruli. It causes inflammation, disrupts kidney function and damages the glomeruli. Renal failure can occur in about half of all patients in as little as 12 years, leading to dialysis or a kidney transplant.

A multi-center clinical trial of avacopan for the treatment of C3G will commence in 2017, ChemoCentryx have confirmed.