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Risankizumab for Psoriasis. BLA filing announced April 25, 2018. PDUFA estimate April 24, 2019. "The risankizumab submission represents an important milestone in our goal of advancing treatment for people living with
Risankizumab for Psoriasis. BLA filing announced April 25, 2018. PDUFA estimate April 24, 2019.
“The risankizumab submission represents an important milestone in our goal of advancing treatment for people living with immune-mediated diseases,” said Michael Severino, M.D., executive vice president, research and development and chief scientific officer, AbbVie. “Risankizumab has the potential to be an important treatment option for people living with plaque psoriasis and we look forward to working with the FDA throughout the review process.”
The BLA is supported by data from the global risankizumab Phase 3 psoriasis program evaluating more than 2,000 patients with moderate to severe chronic plaque psoriasis across four Phase 3 studies: ultIMMA-1, ultIMMa-2, IMMhance and IMMvent.1-3 Across all four studies, risankizumab met all co-primary and ranked secondary endpoints with no new safety signals across the Phase 3 program.1-3 Top-line results of these clinical studies were previously announced in October and December 2017. Risankizumab is not approved by regulatory authorities and its safety and efficacy have not been established.
Risankizumab is part of a collaboration with Boehringer Ingelheim, with AbbVie leading future development and commercialization of risankizumab globally.
Psoriasis is thought to be an immune system problem. Triggers include infections, stress, and cold. The most common symptom is a rash on the skin, but sometimes the rash involves the nails or joints. Three million cases are diagnosed in the U.S. each year.
Praluent (alirocumab) ODYSSEY OUTCOMES for cardiovascular events. PDUFA date for sBLA filing April 28, 2019. Back on September 12, 2018, Regeneron Pharmaceuticals and Sanofi announced that the FDA had accepted a
Praluent (alirocumab) ODYSSEY OUTCOMES for cardiovascular events. PDUFA date for sBLA filing April 28, 2019.
Back on September 12, 2018, Regeneron Pharmaceuticals and Sanofi announced that the FDA had accepted a supplemental Biologics License Application (sBLA) for Praluent® (alirocumab) Injection, a PCSK9 inhibitor. The sBLA outlines a proposed update to the Prescribing Information to include the effect of Praluent in reducing the overall risk of major adverse cardiovascular events (MACE). MACE is an umbrella term that includes heart attack, ischemic stroke, death from coronary heart disease and unstable angina requiring hospitalization. The FDA set a Prescription Drug User Fee Act (PDUFA) action date of April 28, 2019.
The sBLA is supported by data from ODYSSEY OUTCOMES, a Phase 3 cardiovascular outcomes trial that assessed the effect of Praluent in 18,924 patients who had an acute coronary syndrome (ACS), such as a heart attack, between 1-12 months (median 2.6 months) before enrolling in the trial. Results of the ODYSSEY OUTCOMES trial were presented at the American College of Cardiology’s 67th Annual Scientific Session & Expo in March 2018.
The effect of Praluent on cardiovascular morbidity and mortality, including MACE, is currently being reviewed and has not been fully evaluated by any regulatory authority.
In addition, the FDA recently approved an update to the Praluent Prescribing Information to include clinical information regarding its use in patients with heterozygous familial hypercholesterolemia (HeFH) who require additional lowering of LDL-C along with diet and maximally-tolerated statin therapy and who are undergoing apheresis treatment. Apheresis is a procedure where LDL-C is removed from the blood, in a process similar to kidney dialysis. The recommended dose of Praluent in patients undergoing LDL apheresis is 150 mg once every 2 weeks. Praluent can be administered without regard to timing of apheresis.
The update is supported by data from the pivotal Phase 3 ODYSSEY ESCAPE trial of 62 patients with HeFH, an inherited form of high cholesterol, whose cholesterol levels required chronic, weekly or bi-weekly apheresis therapy.
Praluent inhibits the binding of PCSK9 (proprotein convertase subtilisin/kexin type 9) to the LDL receptor and thereby increases the number of available LDL receptors on the surface of liver cells to clear LDL, which lowers LDL-C levels in the blood. Praluent is being developed by Regeneron and Sanofi under a global collaboration agreement and was invented by Regeneron using the company’s proprietary VelocImmune® technology that yields optimized fully-human monoclonal antibodies.
Praluent is approved in more than 60 countries worldwide, including the U.S., Japan, Canada, Switzerland, Mexico and Brazil, as well as the European Union (EU). In the U.S., Praluent is approved for use as an adjunct to diet and maximally tolerated statin therapy for the treatment of adults with HeFH or clinical atherosclerotic cardiovascular disease (ASCVD) who require additional lowering of LDL-C. The effect of Praluent on cardiovascular morbidity and mortality has not been determined.
Regeneron Pharmaceuticals News
OTL-300 for transfusion-dependent beta-thalassemia (TDBT). Phase 1/2 data to be presented April 29, 2019. BOSTON and LONDON, April 15, 2019 (GLOBE NEWSWIRE) -- Orchard Therapeutics (NASDAQ: ORTX), a leading commercial-stage biopharmaceutical
OTL-300 for transfusion-dependent beta-thalassemia (TDBT). Phase 1/2 data to be presented April 29, 2019.
BOSTON and LONDON, April 15, 2019 (GLOBE NEWSWIRE) — Orchard Therapeutics (NASDAQ: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced the achievement of clinical proof-of-concept based on data from the trial of its ex vivo, autologous, hematopoietic stem cell (HSC) gene therapy, OTL-300, for the treatment of transfusion-dependent beta-thalassemia (TDT). The data will be published online today in an abstract via the American Society of Gene & Cell Therapy (ASGCT) website, and the full data set will be featured in an oral presentation at the 22nd ASGCT Annual Meeting to be held on April 29 – May 2, 2019 in Washington, D.C. Specifically, the presentation will report the updated safety and efficacy results of OTL-300 in nine transfusion-dependent severe phenotype TDT patients (six pediatric, three adult), including β0/β0 genotype, with minimum one year post-treatment follow up, as well as details on engraftment of gene-modified cells and vector integration site analyses.
The primary endpoint of transfusion reduction was achieved after one year of follow-up in eight out of nine TDT patients treated with OTL-300. All nine patients met the safety endpoints with no adverse events related to the therapy.
“We are pleased OTL-300 has shown clinical benefit in severe transfusion-dependent beta-thalassemia and has the potential to be a part of a new wave of treatment options for these patients,” said Andrea Spezzi, MBBS, FFPM, chief medical officer at Orchard. “We believe these data support proof-of-concept and the further development of this therapeutic approach. This marks the fifth program from Orchard’s dedicated gene therapy portfolio to achieve clinical proof-of-concept and further supports our belief that a single administration of gene-modified autologous hematopoietic stem cells can lead to durable engraftment and potential disease correction.”
The proof-of-concept study utilized a lentiviral vector containing the human beta-globin gene (GLOBE) and an adapted conditioning regimen. The study also included pediatric patients and patients with a spectrum of TDT genotypes, including the most severe genotype, β0/β0, and patients with notably high pre-treatment transfusion requirements.
Two additional abstracts from Orchard’s clinical and preclinical pipeline were also accepted for presentation at ASGCT. All three accepted abstracts will be published online on the ASGCT website later today.
Presentation details for OTL-300:
Title: Gene Therapy for the Treatment of Adult and Pediatric Patients Affected by Transfusion Dependent Beta-Thalassemia
Presenter:Giuliana Ferrari, San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET) and University Vita-Salute San Raffaele, Milan, Italy
Session: Clinical Gene Therapies for Blood Diseases
Date: Monday. April 29, 2019
Time:10:30 – 10:45 a.m. ET
Location:Washington Hilton, Jefferson room
Abstract number: 49
Beta-thalassemia is a genetic blood disorder caused by a mutation in the beta-globin gene, a fundamental protein required for red blood cells to work correctly. Over 300 mutations in the beta-globin gene are known, which give rise to many different forms of beta-thalassemia, with variable severity. The most damaging mutations cause the almost complete absence of the protein in a patient’s blood, causing them to rely on frequent blood transfusions to survive or a bone marrow transplant from a compatible donor. OTL-300 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy developed for the treatment of TDT that Orchard acquired from GSK in April 2018.
Orchard Therapeutics News
HTX-011 for post operative pain. PDUFA date under priority review April 30, 2019.
“We are pleased to receive Priority Review designation for the HTX-011 NDA,” said Barry D. Quart, Pharm.D., Chief Executive Officer of Heron. “We believe that HTX-011 could have a considerable impact on the lives of patients by significantly reducing the proportion of patients who experience severe pain and receive opioids after surgery, especially at discharge. We look forward to continuing to work closely with the FDA during the review process with the goal of bringing this important product to patients in 2019.”
The FDA had previously granted Breakthrough Therapy designation for HTX-011 based on the results of Phase 2 studies and two completed Phase 3 studies, which showed that HTX-011 produced significant reductions in both pain intensity and the need for opioids through 72 hours post-surgery compared to placebo and bupivacaine solution, the standard-of-care. The FDA has now granted Priority Review designation to the NDA for HTX-011. Priority Review designation is for drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment or prevention of serious conditions. HTX-011 is the first and only non-opioid, long acting local anesthetic to demonstrate in Phase 3 studies a statistically significant reduction in severe pain and an increase in the number of patients who require no opioids for 72 hours postoperatively versus bupivacaine solution, the standard-of-care. The overall safety profile of HTX-011, administered locally into the surgical site without a needle, was similar to that of the well-established safety profile of bupivacaine solution, without evidence of meloxicam-related toxicities.
“Despite ongoing efforts to prevent opioid abuse, patients continue to receive large quantities of opioids for postsurgical pain,” said Jay Redan, M.D., FACS, Medical Director of Minimally-Invasive General Surgery at Florida Hospital Celebration Health. “There is a significant need for safe, effective and non-addictive options that can decrease opioid exposure and improve the patient recovery experience, as well as make an impact on the opioid epidemic by significantly reducing the amount of opioids necessary to take home for pain management.”
HTX-011, which utilizes Heron’s proprietary Biochronomer® drug delivery technology, is an investigational, long-acting, extended-release formulation of the local anesthetic bupivacaine in a fixed-dose combination with the anti-inflammatory meloxicam for the management of postoperative pain. By delivering sustained levels of both a potent anesthetic and a local anti-inflammatory agent directly to the site of tissue injury, HTX-011 was designed to deliver superior pain relief while reducing the need for systemically administered pain medications such as opioids, which carry the risk of harmful side effects, abuse and addiction. HTX-011 has been shown to reduce pain significantly better than placebo or bupivacaine alone in five diverse surgical models: hernia repair, abdominoplasty, bunionectomy, total knee arthroplasty and breast augmentation. HTX-011 was granted Fast Track designation from the FDA in the fourth quarter of 2017 and Breakthrough Therapy designation in the second quarter of 2018. Heron submitted an NDA to the FDA for HTX-011 in October of 2018 and received Priority Review designation in December 2018. The FDA set a Prescription Drug User Fee Act goal date of April 30, 2019.
Heron Therapeutics News
Contepo for complicated urinary tract infections (cUTI). PDUFA date under priority review April 30, 2019. Back on January 14, 2019, Nabriva Therapeutics announced that the FDA had updated the PDUFA (Prescription
Contepo for complicated urinary tract infections (cUTI). PDUFA date under priority review April 30, 2019.
Back on January 14, 2019, Nabriva Therapeutics announced that the FDA had updated the PDUFA (Prescription Drug User Fee Act) goal date for the completion of the FDA’s review of the CONTEPO New Drug Application (NDA) from June 30, 2019 to April 30, 2019. The two-month acceleration is due to a clarification of the classification and subsequent expedited review period for the CONTEPO NDA submitted in October 2018. In addition to priority review, CONTEPO has been granted Qualified Infectious Disease Product (QIDP) and Fast Track designations by the FDA for the treatment of several serious infections, including cUTI.
Urinary tract infections (UTIs) are a significant health problem in both the community- and hospital-based treatment settings. It is estimated that 150 million UTIs occur yearly worldwide, accounting for $6 billion in health care expenditures, according to the American Urological Association. Patients with complicated urinary tract infections (cUTIs), which includes acute pyelonephritis (kidney infection), have pyuria and bacteria in their urine, in association with features such as fever, chills, malaise or flank/back pain, in the setting of a functional or anatomical abnormality of the urinary tract or a history of catheterization. Patients who fail to respond to an initial course of antibiotics may develop a cUTI, which occurs when the bacteria are embedded in the bladder wall or ascend to the kidneys, where they can multiply more slowly and are much harder to address with antibiotics. In most cases, cUTIs occur following treatment for a normal UTI because antibiotics were given too late, for too short a period of time, at too low of a dose course or the wrong antibiotic was used and did not provide adequate spectrum of coverage. An estimated three million cases of cUTIs are treated in the hospital setting in the United States each year for Gram-negative infections. Enterobacteriaceae are the most common pathogens causing cUTIs and, currently, widespread antibiotic resistance limits the effective treatment options for cUTI. Ineffectively managed cUTI can lead to increased treatment failure rates, recurrence of infection, increased re-hospitalization, and increased morbidity and mortality.
CONTEPO™ (fosfomycin for injection, previously referred to as ZTI-01 and ZOLYD) is a novel, potentially first-in-class in the United States, intravenous investigational antibiotic with a broad spectrum of Gram-negative and Gram-positive activity, including activity against most contemporary multi-drug resistant (MDR) strains such as ESBL-producing Enterobacteriaceae. Intravenous (I.V.) fosfomycin has been approved for a number of indications and utilized for over 45 years in Europe to treat a variety of infections, including cUTIs and other serious bacterial infections. CONTEPO utilizes a new dosing approach, originally developed by Zavante (which Nabriva Therapeutics acquired), to optimize its pharmacokinetics and pharmacodynamics. Nabriva Therapeutics believes these attributes, along with the positive clinical experience worldwide, support CONTEPO as a first-line treatment for cUTIs, including acute pyelonephritis, suspected to be caused by MDR pathogens. At least 20 percent of cUTIs are caused by MDR bacteria and limited treatment options are available in the U.S. In addition, non-clinical data have shown that CONTEPO acts in combination with certain other antibiotics to improve bacterial killing.