Methadone insomnia therapy is approved, and Novartis met inhibitor is qualified for priority review

MSD company announced that FDA approved it to update the label information of belsomra (suvorexant), reflecting its clinical data in the treatment of insomnia symptoms in patients with mild to moderate Alzheimer's disease Previously, belsomra has been approved for the treatment of insomnia characterized by difficulty sleeping and / or sleep maintenance Belsomra, a "first in class" orexin receptor antagonist developed by MSD, was approved for marketing in the United States in 2014 to treat insomnia patients Orexin is a neurotransmitter found in specific parts of the brain, which can help people stay awake The data that led to the label update came from a four week, randomized, double-blind, placebo-controlled, three-phase clinical trial designed to assess the safety and efficacy of belsomra in the treatment of 285 patients with mild to moderate insomnia with Alzheimer's disease The results showed that compared with placebo group, belsomra significantly improved the total sleep time (TST) and wake-up after sleep (wasO) indexes Attack frequency significantly reduced! Zogenix new antiepileptic drugs reach the main end point of phase 3 Zogenix, which is committed to the development of rare disease therapy, announced that its oral fluoroamphetamine fintepla (zx008) reached the primary end point in a critical phase 3 trial for Lennox Gastaut syndrome (LGS) Compared with the placebo group, the frequency of seizures in the fintepla group was significantly lower than that at baseline The fintepla developed by zogenix is a liquid form of fluoroamphetamine It can reduce the frequency of epileptic seizures by regulating serotonin mechanism and sigma-1 receptor activity Previously, fintepla was awarded orphan drug qualification by FDA and European drug administration to treat LGS patients At present, the new drug application (NDA) of fintepla for the treatment of epilepsy related to Dravet syndrome has obtained the priority review qualification of FDA, and it is expected to receive the reply before March 25 this year The key phase 3 trial, called study 1601, was divided into two parts The first part aims to evaluate the safety and efficacy of fintepla in current antiepileptic therapy for LGS patients, involving 263 patients aged 2 to 35 years These patients have been treated with one or more antiepileptic drugs, but the disease has not been well controlled Their median baseline frequency of seizures was 77 per month Four weeks after the baseline attack frequency was determined, the randomized patients were adjusted to the treatment dose within the titration period of two weeks, followed by a fixed dose maintenance period of 12 weeks Patients who completed part 1 are eligible for Part 2 clinical trials The second part is a 12-month open label extension study to assess the long-term safety and effectiveness of fintepla ▲ study 1601 reached the main end point (photo source: zogenix official website) The results of this trial showed that the frequency of seizures in LGS patients in the fintepla treatment group was significantly lower than that at baseline, the primary end point of this trial The median frequency of seizures was reduced by 26.5% in the 0.7 mg / kg / day fintepla group compared with 7.8% in the placebo group In addition, patients in the fintepla treatment group also showed improvement at multiple secondary endpoints Myovant Sciences announced that it is in the research of releugolix combination therapy, and it has reached the main efficacy end point in the three-stage expanded clinical study of the treatment of hysteromyoma patients, liberty The data further support the new drug application (NDA) to be submitted in April this year Each tablet of relugolix combination therapy contains relugolix (40 mg), estradiol (1.0 mg), and norethisterone acetate (0.5 mg) As a GnRH receptor antagonist, relugolix can reduce the level of estrogen produced by the ovary, thus alleviating a series of symptoms of hysteromyoma ▲ molecular structure formula of releugolix (picture source: meodipt [public domain]) The results of the liberty extension study showed that the releugolix combination therapy could maintain the BMD of patients and achieve a remission rate of 87.7% in one year (the remission standard was that the amount of menstrual bleeding was less than 80 ml, or the amount of menstrual bleeding was reduced by more than 50% in the last 35 days of the treatment cycle compared with the baseline) The data from this trial validated the persistence mitigation observed in the liberty 1 and 2 trials In addition, the mean amount of menstrual bleeding in the treatment group was 89.9% less than that at baseline The full data of the experiment is expected to be released at a future scientific conference Novartis announced that FDA has accepted a new drug application (NDA) from its met inhibitor capmatinib (inc280) for the treatment of advanced non-small cell lung cancer (NSCLC) patients with mutations in exon 14 of met gene The FDA also granted the application a priority review qualification and is expected to respond within six months If approved, capmatinib would be the first approved treatment for patients with advanced NSCLC carrying a jump mutation in exon 14 of the met gene, the press release said Capmatinib is an oral small molecule met inhibitor with high selectivity, which was initially discovered by Incyte Novartis obtained its R & D and promotion license in 2009 Previously, FDA awarded capmatinib breakthrough therapy certification ▲ capmatinib features (picture source: clinical cancer research) The FDA's priority review was based on a phase 2 clinical trial called geometry mono-1, in which 97 patients with advanced or metastatic NSCLC with a jump mutation in met exon 14 were recruited The results show that capmatinib can bring potential therapeutic effects no matter whether the patients have been treated before or not The total remission rates of capmatinib were 67.9% and 40.6% respectively The median duration of remission was 11.14 months and 9.72 months, respectively Seattle genetics announced that FDA has accepted its new drug application (NDA) for the specific oral small molecule tyrosine kinase inhibitor tucatinib of HER2, which is used in combination with trastuzumab and capecitabine to treat unresectable local late or metastatic HER2 positive breast cancer patients The FDA also granted the application a priority review qualification and is expected to respond by August 20 this year The application will be reviewed in accordance with the FDA real time Oncology Review (RTOR) and the Orbis pilot project RTOR allows FDA to obtain key data of clinical trials before formal application and start to communicate with applicants The project may significantly accelerate the approval process of anticancer drugs The Orbis program aims to help patients in other countries get treatment drugs as early as possible Tucatinib has high selectivity to HER2, but has no obvious inhibitory effect on EGFR belonging to the same family of human epidermal growth factor receptor Previous studies have shown that, whether used as a single therapy or in combination with chemotherapy and other HER2 targeted drugs, tucatinib has shown antitumor activity Previously, the U.S FDA has granted tucatinib orphan drug qualification and breakthrough treatment certification for breast cancer patients The new drug application was submitted based on the results of a phase 3 trial called her2climb The trial is a randomized, double-blind, active control group key clinical study, aimed to compare the efficacy and safety of tucatinib combined with standard treatment drugs trastuzumab and capecitabine in the treatment of locally advanced unresectable or metastatic HER2 positive breast cancer patients compared with trastuzumab and capecitabine Patients in the trial have received trastuzumab, pertuzumab and ADO trastuzumab emtansine (t-dm1), and 47% of patients with brain metastasis ▲ PFS (above) and OS data of tucatinib triple therapy (below) (photo source: New England Journal of Medicine) The results showed that the combination therapy with tcatinib significantly improved the progression free survival (PFS) of patients, and reduced the risk of disease progression or death by 46% compared with the active control group In addition, triple therapy improved overall survival (OS) and reduced the risk of death by 34% compared with the control group In the subgroup of patients with brain metastases, triple therapy also showed excellent PFS, reducing the risk of disease progression or death by 52% Deciphera announced that FDA has accepted its new drug application (NDA) of kit and triprotinib, a PDGFR α kinase inhibitor, for the treatment of advanced gastrointestinal stromal tumor (GIST) patients who have received imatinib, sunitinib and regorafenib At the same time, the FDA granted the application priority review qualification, which is expected to be replied by August 13 this year Currently, FDA is reviewing this according to RTOR pilot project RTOR allows FDA to obtain key data of clinical trials before formal application and start to communicate with applicants The project may significantly accelerate the approval process of anticancer drugs Ripretinib is a kit or PDGFR α kinase inhibitor, which is used to treat kit or PDGFR α driven related cancers, including gist, systemic mastocytosis (SM) and other cancers Ripretinib is specifically designed to improve the treatment of GIST patients by inhibiting broad-spectrum mutations in kit or PDGFR α Previously, the US FDA has granted ripretinib fast track qualification and breakthrough therapy certification for the treatment of advanced GIST patients who have previously been treated with imatinib, sunitinib and regorafenib Molecular structure formula of ripretinib (picture source: PubChem) This NDA submission is based on the positive results achieved by ripretinib in the phase 3 trial Invictus The trial was a randomized, double-blind, placebo-controlled study in which 129 patients were treated with ripretinib or placebo in a 2:1 ratio to assess the efficacy of ripretinib in patients with advanced GIST who had previously been treated with imatinib, sunitinib and regorafenib The results showed that riprotinib reached the primary end point of PFS improvement The PFS in the treatment group was 27.6 weeks, while that in the placebo group was only 4.1 weeks Patients in the treatment group had an 85% lower risk of disease progression or death compared to the placebo group In addition, the objective response rate (ORR) was 9.4% in the treatment group and 0% in the placebo group The total survival time (OS) of the patients in the treatment group was 15.1 months, while that in the placebo group was only 6.6 months, achieving a significant improvement in clinical significance The US FDA approved the launch of the oral lactose alcohol therapy pizensy (lactitol) developed by the Braintree laboratories to treat adult patients with chronic idiopathic constipation (CIC) ▲ molecular structure formula of latex (picture source: FDA) Pizensy is an oral lactulose analogue It is a kind of osmolytic laxative, which can promote the water flow into the intestine, and then achieve the effect of defecation in the colon This approval is based on the results of pizensy's trial in 807 CIC patients, which used the analysis of CSBMs as the end point to evaluate the efficacy of pizensy The results showed that 25% of the patients in the pizensy group achieved CSBMs at least three times in a given week, or from baseline to