What new drugs are expected in the first quarter of 2016?

Source: Xinkangjie on January 13, 2016, three new drugs for the treatment of rare genetic diseases attracted everyone's attention They are eteplirsen, a new drug for Duchenne muscular dystrophy, which will release the results of new drug approval in February 2016, and epidilex, a new drug for the treatment of Dravet syndrome, which will release the results of phase III clinical trials in the first quarter of 2016 And avoralstat, a new drug for hereditary angioneuroedema |1 The new drug eteplirsen eteplirsen is a new antisense diaminomorpholine phosphate oligonucleotide developed by Sarepta therapeutics company in the United States At present, phase IIB clinical trial has been completed for the treatment of Duchenne muscular dystrophy (DMD), a rare hereditary disease Duchenne muscular dystrophy is a primary skeletal muscle necrotic disease characterized by skeletal muscle weakness caused by genetic factors Clinically, it is mainly manifested as progressive skeletal muscle atrophy and weakness, which can affect the heart muscle The disease is associated with mutations in dystrophin If the gene encoding dystrophin is mutated, it will lead to the failure of muscle cells to produce normal dystrophin, and eventually lead to the death of muscle cells At present, there is no effective treatment, mostly steroid hormone and physical therapy to control symptoms Eteplirsen can induce the transcription of dystrophin mRNA to skip exon 51, restore the ORF, and synthesize dystrophin with certain function The U.S FDA has previously granted eteplirsen orphan drug qualification, and also granted fast track approval qualification The FDA's peripheral and central nervous system drug review committee will begin to review eteplirsen's new drug application documents on January 22, 2016 Whether eteplirsen can pass the examination and approval depends on the results of a phase IIB clinical trial (clinical trials.gov Registration No nct01396239) In this clinical trial, 12 patients with Duchenne muscular dystrophy were included The clinical trial results showed that compared with the control group, the disease progress of the patients in the treatment group was delayed, and the 6-meter walking test ability of the patients in the treatment group was significantly improved (16.7% versus 46.2%) Drisapersen, a drug of the same kind developed by bioeplirsen, has not been approved smoothly In November 2015, FDA issued a notice questioning the efficacy and safety of drisapersen in the treatment of DMD The announcement said drisapersen is too toxic even in a fatal and incurable disease like DMD, including thrombocytopenia and severe kidney damage, and there is no reliable evidence for its efficacy Therefore, the prospect of eteplirsen is uncertain It is expected that the US FDA will release the results of new drug approval on February 26, 2016 |2 Epidilex epidilex (gwep1332), a new drug for the treatment of Dravet syndrome, is a child Dravet syndrome treatment drug developed by GW pharmaceutical company, which is currently in phase III clinical trials Dravet syndrome is a rare epileptic encephalopathy with hereditary childhood onset, which is caused by SCN1A gene mutation Most patients almost have cognitive impairment, 50% of them have severe mental retardation, and the early occurrence of myoclonic attack and atypical aphonia attack has a greater impact on cognitive Most of the children with Dravet syndrome have poor efficacy on multiple antiepileptic drugs Epidiolex is a kind of oral liquid preparation, which takes hemp bisphenol as the main active ingredient At present, FDA of the United States has granted epidilex orphan drug qualification GW pharmaceutical company is expected to release the results of a phase III clinical trial named gwep1332 in the first quarter of 2016, and another phase III clinical trial named gwep1424 in 2016 |3 Avoralstat, a new drug for hereditary angioneurotic edema, is a therapeutic drug for hereditary angioedema developed by biocryst pharmaceutical It is currently in phase II / III clinical trial (named opus-2 study) Hereditary angioedema is a rare autosomal dominant genetic disease, also known as C1 esterase inhibitor (C1INH) deficiency Because of the mutation of the gene encoding C1INH, the mutation causes the abnormal function of C1INH in plasma, with or without the decrease of C1INH content C1INH is a natural peptidase inhibitor, which is involved in the regulation of vascular response Avoralstat is an oral peptidase inhibitor, similar to the listed drug ecallantide (dyax), which is also a kallikrein inhibitor In 2009, avoralstat was approved by the US FDA for the treatment of hereditary angioneuroedema But ecallantide (dyax) is a subcutaneous injection At present, American FDA has awarded avoralstat orphan drug qualification If the results of phase III clinical trials of avoralstat are excellent, it will be the first drug to be given orally to treat hereditary angioneurotic edema.