AstraZenecas Farxiga pads its case for new heart-helping approval

FiercePharma | August 20, 2019

AstraZeneca is still stinging after its SGLT2 diabetes drug Farxiga was waved off by the FDA in Type 1 disease. But heres something that could ease the pain- New outcomes data showing Farxiga cut cardiovascular risks in heart failure patients with or without diabetes. In a phase 3 trial, Farxiga cut the risk of cardiovascular death or hospitalization in patients with reduced ejection fraction HFrEF, when added to standard treatments for heart failure. The data could help Farxiga score a CV risk-reduction approval, a distinction its rivals in the SGLT2 class already have. The trial, dubbed Dapa-HF, was the first to test an SGLT2 drug against heart failure in patients with or without Type 2 diabetes, the company said. It pitted Farxiga plus standard therapy against standard therapy alone. AstraZeneca plans to take the data to regulators "as soon as possible," Mene Pangalos, EVP of BioPharmaceuticals R&D, said in a statement.

Spotlight

The development of novel therapeutic products involves a complex network of internal departments and external partners. Different groups across Process Development organizations typically use a variety of approaches to capture, manage, analyze and report data. These can range from paper and Excel to in-house developed electronic systems or commercial solutions. Regardless of their IT maturity, the lack of a consistent approach has a negative impact on the agility of the business including.

Spotlight

The development of novel therapeutic products involves a complex network of internal departments and external partners. Different groups across Process Development organizations typically use a variety of approaches to capture, manage, analyze and report data. These can range from paper and Excel to in-house developed electronic systems or commercial solutions. Regardless of their IT maturity, the lack of a consistent approach has a negative impact on the agility of the business including.

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BUSINESS INSIGHTS

Stevanato Group and Gerresheimer AG Announce Collaboration on the Development of an Innovative Ready-To-Use Vial Platform

Gerresheimer and Stevanato Group | September 08, 2022

Stevanato Group S.p.A. a global provider of drug containment, drug delivery and diagnostic solutions to the pharmaceutical, biotechnology and life sciences industries, and Gerresheimer AG, a leading global provider of healthcare & beauty and drug delivery systems for pharma, biotech and cosmetics, announce that they have jointly developed a high-end Ready-To-Use (RTU) solution platform with an initial focus on vials, based on Stevanato Group’s market-leading EZ-fill® technology. This collaboration is projected to help customers gain efficiencies, improve the quality standard, increase speed to market, reduce total cost of ownership (TCO) and help mitigate supply chain risk. Stevanato Group and Gerresheimer AG are addressing rising demand for RTU vials in the market and the partnership serves as a market enabler to fully support customers’ evolving needs and establish a gold standard in the industrial filling process. The collaboration aims to make RTU vials a standard, available to a wide number of pharma companies globally – offering premium quality solutions to patients and reducing complexity for pharma operations. The new solution will be available to other players in the market to standardize fill-finish operations from early phase drug development to commercialization. The new RTU solution platform from Stevanato Group and Gerresheimer AG will share the same secondary packaging, production process and sterilization method, ensuring consistent available capacity and a reliable double sourcing to the pharmaceutical industry. A hallmark of the new RTU platform is a significant reduction in particles, improving the overall quality and performance of the RTU solutions. In addition, customers can count on a high-end solution made by two major players in the drug containment industry with a long history of innovation. The new jointly developed vial platform and trademark will be presented in detail to the market at the CPhI Worldwide in Frankfurt in November 2022. “Our EZ-fill® vial platform is the market’s most established choice for pre-sterilized containers, and this enhanced version for RTU vials will bring to the market its full potential in terms of quality, flexibility and value for Pharmaceutical Companies and CMOs. Through our collaboration with Gerresheimer AG we are responding to market demand, and we expect to achieve another important milestone in creating a reliable ecosystem to support, accelerate and de-risk the conversion from bulk to EZ-fill® vials already underway in the market.” Mauro Stocchi, Chief Business Officer at Stevanato Group “The new developed RTU vial platform stands out in terms of quality, TCO and sustainability and takes the use of RTU solutions like vials and, in the future, cartridges to a new level,” said Dr. Lukas Burkhardt, Member of the Management Board of Gerresheimer AG. "Due to the strong reduction of the particle load, the quality is significantly increased. Our new innovative solution will convince the market to significantly accelerate the conversion from bulk to RTU vials.” About Stevanato Group Founded in 1949, Stevanato Group is a leading global provider of drug containment, drug delivery and diagnostic solutions to the pharmaceutical, biotechnology and life sciences industries. The Group delivers an integrated, end-to-end portfolio of products, processes and services that address customer needs across the entire drug life cycle at each of the development, clinical and commercial stages. Stevanato Group’s core capabilities in scientific research and development, its commitment to technical innovation and its engineering excellence are central to its ability to offer value added solutions to clients. About Gerresheimer AG Gerresheimer AG is the global partner for pharmaceutics, biotech, healthcare, and cosmetics with a very broad product range for pharmaceutical and cosmetic packaging solutions and drug delivery systems. The company is an innovative solution provider from concept to delivery of the commercial product. Gerresheimer achieves its ambitious goals through a high level of innovative strength, industrial competence and concentration on quality and customer focus. In developing innovative and sustainable solutions, Gerresheimer relies on a comprehensive international network with numerous innovation and production centers in Europe, America and Asia. Gerresheimer produces close to its customers worldwide with around 11,000 employees and generated annual revenues in 2021 of around EUR 1.5b. With its products and solutions, Gerresheimer plays an essential role in people's health and well-being.

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PHARMACY MARKET

Acorda Therapeutics Enters into License Agreement with Asieris Pharmaceuticals

Asieris | August 30, 2022

Acorda Therapeutics, Inc. announced that it has entered into a license agreement relating to its preclinical asset, Nepicastat, with Asieris Pharmaceuticals, a biotechnology company headquartered in China. Under the terms of the agreement, Acorda will receive an upfront payment of $500,000, and up to an additional $7 million based on the achievement of regulatory milestones. Acorda will also receive a royalty on future net sales. Nepicastat is a small molecule drug and the license agreement provides for its development for all non-psychiatric indications and therapeutic uses. The asset has been held by Acorda’s U.S. subsidiary, Biotie Therapies, Inc. About Acorda Therapeutics Acorda Therapeutics develops therapies to restore function and improve the lives of people with neurological disorders. INBRIJA® is approved for intermittent treatment of OFF episodes in adults with Parkinson’s disease treated with carbidopa/levodopa. INBRIJA is not to be used by patients who take or have taken a nonselective monoamine oxidase inhibitor such as phenelzine or tranylcypromine within the last two weeks. INBRIJA utilizes Acorda’s innovative ARCUS® pulmonary delivery system, a technology platform designed to deliver medication through inhalation. Acorda also markets the branded AMPYRA® Extended Release Tablets, 10 mg. About Asieris Asieris Pharmaceuticals founded in March 2010, is a global biopharma company specializing in discovering, developing and commercializing innovative drugs for the treatment of genitourinary tumors and other related diseases. We strive to improve human health and help people live a more dignified life. We aim to become a global pharma leader that integrates R&D, manufacturing and commercialization in our areas of focus, as we provide best-in-class integrated diagnosis and treatment solutions for patients in China and worldwide.

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PHARMACY MARKET

Alnylam Announces FDA Approval of Supplemental New Drug Application for OXLUMO® (lumasiran) in Advanced Primary Hyperoxaluria Type 1

Alnylam Pharmaceuticals, Inc. | October 07, 2022

Alnylam Pharmaceuticals, Inc. the leading RNAi therapeutics company, announced that the U.S. Food and Drug Administration approved a label expansion for OXLUMO® an RNAi therapeutic administered via subcutaneous injection, now indicated for the treatment of primary hyperoxaluria type 1 to lower urinary oxalate and plasma oxalate levels in pediatric and adult patients. The approval is based on positive efficacy and safety results of the ILLUMINATE-C Phase 3 study of OXLUMO in patients with severe renal impairment, including those on hemodialysis. PH1 is an ultra-rare genetic disease characterized by oxalate overproduction in the liver. The excess production of oxalate results in the deposition of calcium oxalate crystals in the kidneys and urinary tract and can lead to the formation of painful and recurrent kidney stones and nephrocalcinosis, which can progress to kidney failure. PH1 can also lead to oxalate deposition in multiple organs beyond the kidney, a condition known as systemic oxalosis. The label expansion for OXLUMO is based on positive six-month results from ILLUMINATE-C, where treatment with lumasiran resulted in a substantial reduction in POx from baseline to Month Six in both dialysis-independent and -dependent patients. Patients in both cohorts had a reduction in POx as early as Month One. In Cohort A treatment with OXLUMO led to a 33% least squares (LS) mean reduction in POx from baseline to Month Sixand in Cohort B, treatment with OXLUMO led to a 42% LS mean reduction in POx from baseline to Month Six. OXLUMO demonstrated an encouraging safety and tolerability profile with no deaths or drug-related serious adverse events among enrolled patients. There were two treatment discontinuations due to adverse events in the extension period of the study, neither of which was drug related. The most common AE related to lumasiran was injection-site reaction reported in five of 21 patients which were all mild and transient. “The significance of the label expansion of OXLUMO cannot be overstated, as this milestone provides crucial reassurance among a patient population with the highest unmet need, as well as their caregivers and loved ones, that OXLUMO is available in the US for patients living with PH1, including those with advanced disease. We are grateful to Alnylam for their continued commitment to the PH1 community and for ensuring that all patients — no matter how severe their disease — have the same opportunity to potentially benefit from OXLUMO treatment.” Kim Hollander, Executive Director of the Oxalosis and Hyperoxaluria Foundation In November of 2020, OXLUMO was approved by the FDA for the treatment of PH1 to lower UOx levels in pediatric and adult patients. It was also approved by the European Medicines Agency (EMA) for the treatment of PH1 in all age groups. The Committee for Medicinal Products for Human Use (CHMP) of the EMA delivered a positive opinion recommending variation to the marketing authorization of OXLUMO based on ILLUMINATE-C data from patients with advanced PH1 in September 2022. Lumasiran is also being evaluated in a global Phase 2 study as an investigational treatment in patients with recurrent kidney stone disease and elevated UOx levels. About OXLUMO OXLUMO® is an RNAi therapeutic targeting hydroxyacid oxidase 1. HAO1 encodes glycolate oxidase. Thus, by silencing HAO1 and depleting the GO enzyme, OXLUMO inhibits production of oxalate – the metabolite that directly contributes to the pathophysiology of PH1. OXLUMO utilizes Alnylam’s Enhanced Stabilization Chemistry GalNAc-conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index. OXLUMO has received regulatory approvals from the U.S. Food and Drug Administration for the treatment of primary hyperoxaluria type 1 to lower urinary and plasma oxalate levels in pediatric and adult patients and from the European Medicines Agency for the treatment of PH1 in all age groups. In the pivotal ILLUMINATE-A study, OXLUMO was shown to significantly reduce levels of urinary oxalate relative to placebo, with the majority of patients reaching normal or near-normal levels. In the ILLUMINATE-B pediatric Phase 3 study, OXLUMO demonstrated an efficacy and safety profile consistent to that observed in ILLUMINATE-A. In the ILLUMINATE-C study, OXLUMO resulted in substantial reductions in plasma oxalate in patients with advanced PH1. Across all three studies, injection site reactions were the most common drug-related adverse reaction. OXLUMO is administered via subcutaneous injection once monthly for three months, then once quarterly beginning one month after the last loading dose at a dose based on actual body weight. For patients who weigh less than 10 kg, ongoing dosing remains monthly. About Alnylam Pharmaceuticals Alnylam has led the translation of RNA interference into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding 20 years ago, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam’s commercial RNAi therapeutic products are ONPATTRO® GIVLAARI® OXLUMO® AMVUTTRA® and Leqvio® being developed and commercialized by Alnylam’s partner, Novartis. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P5x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA.

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