Stannsoporfin

It’s a busy week on the U.S. Food and Drug Administration (FDA)’s calendar, although the agency got ahead of itself and approved three of the applications early. Here’s a look. It’s a busy week on the U.S. Food and Drug Administration (FDA) ’s calendar, although the agency got ahead of itself and approved three of the applications early. Here’s a look. Agios Pharmaceuticals had a Prescription Drug User Fee Act (PDUFA) action date of Tuesday, August 21, for its ivosidenib (AG-120) for patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with an isocitrate dehydrogenase 1 (IDH1) mutation. However, the FDA approved this application early, on July 20. The FDA granted the drug Priority Review. The drug is a first-in-class, oral, targeted inhibitor of mutant IDH1. In a related note, Abbott submitted a Premarket Approval (PMA) application for an IDH1 assay on the Abbott 2000 RealTime System, an automated sample preparation and batch analyzer system. Agios and Abbott signed a deal in 2014 where Abbott was responsible for developing and commercializing a RealTime PCR assay for IDH1 mutations in bone marrow and blood, which will act as a companion diagnostic for ivosidenib. Mallinckrodt Pharmaceuticals has a target action date of Wednesday, August 22 for stannsoporfin for the treatment of neonates at risk for developing severe jaundice (hyperbilirubinemia). The drug received Fast Track status. The drug is a heme oxygenase inhibitor that has the potential to cut the production of bilirubin in infants at risk for jaundice. Kala Pharmaceuticals has a target action date of Friday, August 24 for Inveltys (KPI-121 1%), a topical twice-a-day treatment for inflammation and pain in patients after ocular surgery. If approved, it will be the first twice-daily ocular corticosteroid for the treatment of postoperative ocular inflammation. All the others are approved four times a day. The compound uses the company’s proprietary Mucus Penetrating Particle (MPP) technology. Also Friday, August 24, Merck & Co . and Eisai were expecting a response to their supplemental New Drug Application (sNDA) for lenvatinib for the potential first-line treatment of unresectable hepatocellular carcinoma (HCC). This date was an extension from the original date of May 24, as the FDA wanted more time to review the application. They approved the drug a week early, on Friday, August 16. The drug, marketed as Lenvima, is approved in the U.S. for locally recurrent or metastatic, progressive, radioactive iodine-refractory differentiated thyroid cancer. It has also been approved in the U.S. in combination with everolimus in patients with advanced renal cell carcinoma after one prior anti-angiogenic therapy. Friday, August 24 is a busy day for the agency. Regeneron Pharmaceuticals is waiting on a supplemental Biologics License Applications (sBLA) for use of Praluent (alirocumab) for use with apheresis. Praluent is a PCSK9 inhibitor for high cholesterol. Apheresis is a nonsurgical procedure that removes low-density lipoprotein (LDL) cholesterol from a patient’s blood. Shionogi & Co had a PDUFA date of Sunday, August 26 for its NDA for lusutrombopag for treatment of thrombocytopenia in patients with chronic liver disease at increased risk for bleeding associated with invasive procedures. The FDA approved the drug on August 1. The submission was based on two Phase III clinical trials that showed the drug met the pre-specified primary and all key secondary endpoints. The drug also had Priority Review status. Lusutrombopag is an oral, small molecule agonist of the human thrombopoietin receptor. It was approved in 2015 in Japan for the same indication. Shire also has a PDUFA date of Sunday, August 26. In Shire’s case, it’s for lanadelumab’s BLA for angioedema attacks in patients 12 years and older with the rare, genetic disorder, hereditary angioedema. The FDA also granted the drug Priority Review. Lanadelumab is a fully human monoclonal antibody that specifically binds and inhibits plasma kallikrein.

August 9, 2017 By Alex Keown , BioSpace.com Breaking News Staff STAINES-UPON-THAMES, England– At least two companies are beginning to nibble on the bait of Mallinckrodt PLC ’s generics business a few months after the company announced it was exploring the possible divestment. A CNBC-TV report posted on moneycontrol.com indicates that two India-based businesses, Aurobindo Pharma and Intas are interested in acquiring the generics business . The U.K.-based company could garner up to $2 billion for the generics operations. If one of these two companies bites, CNBC said it will mark the biggest ever overseas acquisition for any Indian drug maker. Mallinckrodt announced in May that it was exploring the sale as a way to pivot toward its higher-margin branded drugs. The company has a long history with generics drugs, but branded pharmaceuticals comprises the bulk of its revenues, Reuters noted. But the company’s generics business has been “a drag” on Mallinckrodt, particularly because some of its products “include opiate-based pain killers, which have fallen out of favor with doctors due to their addictive potential,” Reuters said in May. Mallinckrodt’s generics business saw revenue losses of about 18 percent, with sales declining approximately $1 billion. Mallinckrodt’s branded drugs climbed more than 40 percent to $2.3 billion. While rumors swirl that the two Indian companies are in competition for Mallinckrodt’s generics business, the U.K. company has not provided any confirmation, according to the reports. Neither Aurobindo nor Intas provided any additional confirmation. Aurobindo is India’s fourth-largest drugmaker by sales. CNBC noted that Mallinckrodt’s generics business will fit in well with Aurobindo’s existing offerings. While Mallinckrodt is exploring the divestment of its generics business, the company has been filling out its pipeline. Most recently, the company announced it was acquiring privately-held InfaCare , a Pennsylvania-based company focused on therapies for neonatal and pediatric patient. The deal worth up to $425 million will provide Mallinckrodt with InfaCare’s stannsoporfin, a heme oxygenase inhibitor, used to treat elevated bilirubin levels that can cause severe jaundice in infants. In addition to the acquisition, Mallinckrodt has also picked up some momentum from regulatory agencies. In July, the U.S. Food and Drug Administration designated Mallinckrodt’s Stratagraft skin-tissue replacement with the new designation of Regenerative Medicine Advanced Therapy. The company’s investigational, tissue-based therapy is currently under evaluation in a Phase III trial to assess its efficacy and safety in the promotion of autologous skin regeneration of complex skin defects due to thermal burns that contain intact dermal elements (also known as deep partial thickness burns). Also in July, the FDA granted Mallinckrodt’s experimental Duchenne muscular dystrophy drug , MNK-1411, with the Orphan Drug Designation. MNK-1411 (cosyntropin injection) is a depot formulation of tetracosactide, a synthetic 24 amino acid melanocortin receptor agonist. It is expected to begin Phase II trials later this year.