shire plc (SHPGF) Key Developments
FDA Accepts Filing the New Drug Application for Shire's Lifitegrast and Grants Priority Review Designation
Apr 9 15
Shire plc announced that the United States Food and Drug Administration (FDA) has accepted for filing the New Drug Application (NDA) for lifitegrast and granted a Priority Review designation. Lifitegrast is an investigational treatment for dry eye disease in adults and, if approved, has the potential to be the first treatment indicated to address both signs and symptoms of the disease. The FDA is expected to provide a decision on October 25, 2015, based on the Prescription Drug User Fee Act V action date. The FDA grants Priority Review designation to drugs that have the potential to provide significant improvements in the safety or effectiveness for the treatment, diagnosis or prevention of a serious disease. Drugs with Priority Review designation have an accelerated review target of eight months, instead of the standard of 12 months.
Shire plc Reports Topline Results from Phase 2 IMAGO Study
Apr 9 15
Shire plc announced that the 13-week Phase 2 IMAGO trial of its investigational compound SHP625 (LUM001) did not meet the primary or secondary endpoints in the study of 20 pediatric patients with Alagille syndrome (ALGS), a rare, life-threatening genetic disorder that presents with chronic cholestasis (accumulation of bile acids in the liver) and severe pruritus (itching). The primary endpoint was the change from baseline in serum bile acid levels as compared to placebo. The secondary endpoint of pruritus was assessed using the novel ItchRO™ instrument. Mean serum bile acid levels and pruritus at the end of the study were lower in both SHP625 and placebo treated groups as compared to baseline. However, in a post-hoc analysis, a positive correlation between changes from baseline in serum bile acid levels and pruritis was observed in the SHP625 treated group. The number of patients in the placebo treated group was too small to make an accurate assessment of this relationship. There were no treatment emergent serious adverse events in this study. As expected, the most common adverse events were diarrhea and abdominal pain, which were more frequent with SHP625 than with placebo. In addition to IMAGO, two larger placebo-controlled phase 2 studies in ALGS are in progress, one of which has pruritus as the primary endpoint. SHP625 is also being studied in progressive familial intrahepatic cholestasis, primary biliary cirrhosis and primary sclerosing cholangitis.
Shire plc Announces Clear Regulatory Path Forward for SHP465, an Investigational Treatment for Adults with ADHD
Apr 7 15
Shire plc announced that it has reached an agreement with the U.S. Food and Drug Administration on a clear regulatory path for SHP465 (triple-bead mixed amphetamine salts - MAS), an investigational oral stimulant medication being evaluated as a potential treatment for Attention-Deficit/Hyperactivity Disorder (ADHD) in adults. The company has agreed with the FDA to conduct a short-term efficacy and safety study in pediatric patients with ADHD (ages 6-17). While Shire intends to pursue an adult indication, the FDA is requesting this additional pediatric data to better understand the potential effects of SHP465 on children with ADHD in the event of use in this population. The company anticipates the clinical trial's first patient, first visit to take place in August 2015, with study completion targeted by the last quarter of 2016. Shire then expects to submit to the FDA by second quarter 2017 a Class 2 resubmission for approval of SHP465 as a treatment for ADHD in adults, which typically entails a 6-month review. Pending FDA approval, Shire anticipates launching the medicine in the second half of 2017. This update follows Shire's announcement on October 9, 2014, that it was engaging the FDA to determine the parameters of clinical data requirements in order to submit the Class 2 resubmission. Adult patients with ADHD represent the fastest growing segment of the overall ADHD patient population. Data from IMS Health (a global healthcare information and technology firm) suggest that about 10% of adult patients are adding an immediate release medicine to their extended release medicine, most often to gain a longer duration of treatment effect. SHP465 demonstrated a statistically significant difference versus placebo at 16 hours post dosing, with onset of action starting 4 hours post dosing, as measured by the Permanent Product Measure of Performance (PERMP). Common adverse reactions in SHP465 registration trials (incidence =5% and at a rate twice placebo) in adults were: insomnia, decreased appetite, dry mouth, decreased weight, heart rate increased, and dysmenorrhea. These adverse events are generally known to be associated with the use of amphetamine products. There are patents supporting Shire's overall ADHD franchise in the U.S. that extend to 2029. With a launch planned for the second half of 2017, Shire expects that SHP465, following FDA approval, will have three years of Hatch-Waxman exclusivity and at least three patents listed in the FDA Orange Book expiring as late as May 2029. Vyvanse (lisdexamfetamine dimesylate), which has patents expiring in 2023, is a prescription medicine used for the treatment of ADHD in patients 6 years and above and for the treatment of moderate to severe Binge Eating Disorder (B.E.D.) in adults. Vyvanse is not for weight loss. It is not known if Vyvanse is safe and effective for the treatment of obesity. Shire announced on June 12, 2014 that it has agreed to a Written Request by the FDA to conduct pediatric clinical studies to investigate the potential use of Vyvanse for the treatment of ADHD in preschool-age children, ages 4 to 5. Upon FDA confirmation of a timely submission and review of data that adheres to the requirements of the Written Request, Shire will be entitled to the benefits of the Best Pharmaceuticals for Children Act, including a six-month extension to the exclusivity afforded by Shire's patents for Vyvanse.
Shire plc Announces Availability of its Natpara (Parathyroid Hormone) for Injection in the U.S
Apr 1 15
Shire plc announced that Natpara (parathyroid hormone) for injection is now available in the United States. The U.S. Food and Drug Administration (FDA) approved Natpara as an adjunct to calcium and vitamin D to control hypocalcemia in patients with hypoparathyroidism on January 23, 2015. Because of the potential risk of osteosarcoma, Natpara is recommended only for patients who cannot be well-controlled on calcium supplements and active forms of vitamin D alone. Natpara was not studied in patients with hypoparathyroidism caused by calcium-sensing receptor mutations or in patients with acute post-surgical hypoparathyroidism. Natpara will be available through a Risk Evaluation and Mitigation Strategy (REMS) Program and a limited network of specialty pharmacies. Eligible patients who are prescribed Natpara will have access to patient support services through NPS Advantage. These services include access to NPS Advantage Care Coordinators who can provide: information about Natpara, insurance authorization, appeals and financial assistance; assistance with ordering products; and, connect patients with a Nurse Educator. Natpara may cause serious side effects, including possible bone cancer (osteosarcoma). During animal drug testing, Natpara caused some rats to develop a bone cancer called osteosarcoma. It is not known if people who take Natpara will have a higher chance of getting osteosarcoma. See below for additional Important Safety Information and full prescribing information about Natpara.
Shire, AstraZeneca And Roche Show Interest In BioMarin
Mar 30 15
After several media outlets reported that Shire plc (LSE:SHP), AstraZeneca PLC (LSE:AZN) and Roche Holding AG (SWX:ROG) are considering buying BioMarin Pharmaceutical Inc. (NasdaqGS:BMRN), SunTrust says that it would be logical for several drug companies looking to build or expand their orphan disease franchises to buy BioMarin.