FDA advisors reject one Daiichi Sankyo cancer drug, back another

Daiichi Sankyo had a day of mixed fortunes after advisers to the US regulator voted against approval for its leukaemia drug quizartinib, but recommended pexidartinib for a rare tumour. Experts from the FDA’s Oncologic Drugs Advisory Committee (ODAC) met to discuss clinical data from the two drugs, and made non-binding recommendations ahead of the decision date set for them.
While these are only recommendations, the FDA usually follows the advice of its advisory committees. The Japanese pharma is seeking approval for quizartinib to treat patients with relapsed/refractory FLT3-ITD acute myeloid leukaemia (AML). But the committee voted against approval by eight votes to three, unconvinced by the results from the phase 3 QuANTUM-R study. Committee members agreed with FDA reviewers who raised concerns that although the study hit its target of a statistically significant improvement in overall survival, the study design may have biased the results. This was because there was a much higher rate of stem cell transplants in the quizartinib arm, than in a chemotherapy control arm.

Spotlight

Spotlight

Related News

PHARMA TECH

Exclusive Agreement: Specialised Therapeutics & Ascendis Pharma to Distribute Three Endocrinology Therapies in Australia & Select SE Asia Countries

PR Newswire | January 08, 2024

Independent biopharmaceutical company Specialised Therapeutics Asia Pte Ltd (ST) has added three new endocrinology therapies to its specialist portfolio, following an exclusive distribution agreement with Danish company Ascendis Pharma A/S. Under the terms of the agreement, ST will commercialise Ascendis Pharma's weekly injectable paediatric human growth hormone treatment SKYTROFA (lonapegsomatropin), hypoparathyroidism treatment YORVIPATH (palopegteriparatide) and investigational achondroplasia therapy TransCon CNP (navepegritide). The agreement spans ST's key regions of Australia, New Zealand, Singapore, Malaysia, Brunei, Thailand, and Vietnam. Two of the products included in this agreement are already internationally approved: Once-weekly SKYTROFA is a human growth hormone (hGH) approved in the United States for the treatment of paediatric patients aged >1 years weighing >11.5 kg with growth failure due to inadequate secretion of endogenous growth hormone (GH)1 and in the European Union for growth failure in children and adolescents aged from 3 to 18 years due to insufficient endogenous growth hormone secretion (growth hormone deficiency [GHD]). YORVIPATH is a first-in-class parathyroid hormone (PTH) replacement therapy to treat chronic hypoparathyroidism, a rare and potentially serious condition where the body produces no or abnormally low levels of PTH. It is approved in the European Union for the treatment of adults with chronic hypoparathyroidism. The third product – TransCon CNP – is in development by Ascendis Pharma for the treatment of achondroplasia (ACH), the most common genetic form of skeletal dysplasia and resulting disproportionate short stature, following successful Phase 2 trial results. Australian endocrinologist Dr Veronica Preda noted that YORVIPATH would be the first specialist therapeutic option for Australian patients living with hypoparathyroidism. "Hypoparathyroidism can seriously impact quality of life and has potentially life-threatening consequences," Dr Preda said. "To have an option that is able to treat the underlying cause of the disease, moving beyond standard oral calcium and active Vitamin D, is a great step forward." Announcing the partnership, ST Chief Executive Officer Carlo Montagner said this agreement was an important company milestone, signalling ST's expansion into both endocrinology and paediatric medicine. Mr Montagner commented: "We are delighted to have been selected as Ascendis Pharma's exclusive partner for commercialising their portfolio in Oceania and these South-East Asia countries and look forward to launching these critical endocrinology products in our regions as soon as possible. "All three products are valuable inclusions to our broad therapeutic pipeline and our international business, as we continue to leverage our substantial experience commercialising specialist medicines across multiple regions. "We look forward to working with endocrinologists across our territories to make these endocrine therapies available to all eligible patients who may benefit." Ascendis Pharma Executive Vice President and Chief Commercial Officer Camilla Harder Hartvig said ST had been selected to launch the endocrinology portfolio in these countries based on its strong track record commercialising specialist products in multiple regions. "We are delighted to partner with Specialised Therapeutics to broaden the reach of our endocrinology rare disease portfolio, contributing to our shared goal of making a meaningful difference for patients facing unmet medical needs," she said. About Specialised Therapeutics Founded in 2007, Specialised Therapeutics is the region's largest independent specialty pharmaceutical company, providing new therapies and technologies to patients in Australia, New Zealand and across Southeast Asia. Headquartered in Singapore, ST partners with global pharmaceutical, biotech and diagnostic companies to bring novel healthcare opportunities to patients who are impacted by a range of diseases. ST has built a strong track record of success, navigating complex regulatory, reimbursement and commercialisation environments in its diverse regions. The ST mission is to provide specialty therapies where there is an unmet need. The company's broad therapeutic portfolio currently includes novel agents in oncology, haematology, CNS, neurology, endocrinology, ophthalmology and supportive care, although it is not confined to these areas. ST is a member of the World Orphan Drug Alliance (WODA).

Read More

PHARMA TECH

Performance Validation Joins ValGenesis Partner Program as Service Partner for the U.S. Market

PR Newswire | January 24, 2024

ValGenesis Inc., the market leader in enterprise validation lifecycle management systems (VLMS), and Performance Validation, Inc. a 100% employee-owned company serving the life sciences industry since 1988, have partnered to deliver unique compliance-focused digital validation solutions for life sciences companies in the US. Performance Validation is an industry leader with over 30 years of experience in analyzing, identifying, and testing the most critical elements of the manufacturing process for FDA-regulated industries. The company provides environmental mapping, commissioning, qualification, computer system validation, and quality system consulting services. ValGenesis is the industry innovator of digitized validation. The company's platform, which includes its flagship VLMS offering, is used in over 100,000 GMP systems worldwide, helping life sciences companies advance their digital transformation strategy, enforce compliance, and enable standardization with 100% digital, risk-based validation software. In this partnership, Performance Validation will implement and manage next-generation digital validation tools developed by ValGenesis, including ValGenesis VLMS and VLMS Express, a simplified cloud-based digital validation solution for startups and midsized companies. ValGenesis and Performance Validation are providing an integrated governance approach to further strengthen risk and compliance across their customers' manufacturing processes. "We are excited to partner with Performance Validation. Their extensive experience and our industry benchmarked digital validation platform can help life sciences companies implement their digitization plans efficiently according to Industry 4.0 standards," says Bo Olsen, ValGenesis' SVP of Partners. "By establishing a clear strategy that addresses their risk and compliance implications upfront, we can assure them of true business transformation." "Performance Validation is thrilled to embark on this collaborative journey with ValGenesis, leveraging our extensive experience and knowledge of digital validation solutions. It's a powerful combination – our hands-on validation experience and their innovative digital tools. For our clients in the life sciences, this means more efficient, streamlined, and compliant solutions. We're not just adapting to Pharma 4.0; we're helping our clients lead the way in digital transformation," says Brad Henry, Vice President of Service Lines at Performance Validation. ABOUT PERFORMANCE VALIDATION Performance Validation (PV) is a global validation partner for pharmaceutical and medical device manufacturers. Headquartered in Indianapolis, IN, we specialize in turning compressed timelines into compliant ones using innovative, adaptive approaches that balance production realities with strict regulatory requirements. Our best-in-class, cGMP-compliant services cover diverse needs from fully managed CQV to on-demand temperature mapping, smoke studies, software assurance, and more. With a dedicated team consisting of more than 95% engineers, we work closely with regulators and equipment suppliers to keep validation ahead of production curves and keep quality moving forward. ABOUT VALGENESIS ValGenesis, Inc. is the creator of an innovative software platform that serves as a foundation for managing compliance-based validation activities in life sciences companies. ValGenesis, Inc. is the provider of the first enterprise application that manages the corporate validation lifecycle process. This solution is fully compliant with U.S. FDA 21 CFR Part 11 and Annex 11 requirements. As the first fully paperless solution for electronic management of validation execution and approval, ValGenesis was selected by an industry peer review committee to receive the Parenteral Drug Association (PDA) New Innovative Technology Award in 2005.

Read More

PHARMA TECH, PRACTICE MANAGEMENT

Lilly Launches End-to-End Digital Healthcare Experience through LillyDirect

PR Newswire | January 05, 2024

Eli Lilly and Company today announced LillyDirect, a new digital healthcare experience for patients in the U.S. living with obesity, migraine and diabetes. LillyDirect offers disease management resources, including access to independent healthcare providers*, tailored support, and direct home delivery of select Lilly medicines through third-party pharmacy dispensing services. "A complex U.S. healthcare system adds to the burdens patients face when managing a chronic disease. With LillyDirect, our goal is to relieve some of those burdens by simplifying the patient experience to help improve outcomes," said David A. Ricks, Lilly's chair and CEO. "LillyDirect offers more choices in how and where people access healthcare, including a convenient home delivery option to fill Lilly medicines they have been prescribed." For patients living with obesity, migraine and diabetes, LillyDirect offers: LillyDirect Pharmacy Solutions, a digital pharmacy for select Lilly medicines powered by third party online pharmacy fulfillment services. LillyDirect Pharmacy Solutions gives patients a simple option to get consistent access to Lilly medicines they have been prescribed with the added convenience of home delivery. By obtaining medicines directly from Lilly, patients can easily access Lilly's affordability solutions and savings card opportunities are automatically applied for patients who qualify. Medicines fulfilled by pharmacy service providers through LillyDirect Pharmacy Solutions ship free of charge. Disease state and healthcare educational information to help empower and support patients on their care journeys. Access to independent telehealth providers that could complement a patient's current primary care team or be an alternative to in-person care for certain conditions. An independent search tool that allows a patient to find healthcare professionals near them if they prefer in-person care. "We know that people have come to depend on the efficiency and convenience of digital solutions to meet a variety of their everyday needs – healthcare being one of them," said Frank Cunningham, group vice president, global value and access at Lilly. "We launched LillyDirect with the hope that it will offer patients an innovative end-to-end experience to manage their health and access their medicines, so they can get back to living their lives." LillyDirect will continue to be updated and expanded to improve the customer experience. Future updates may include new products, partners and services, such as programs designed to help patients with adherence to their medicines. About Lilly Lilly unites caring with discovery to create medicines that make life better for people around the world. We've been pioneering life-changing discoveries for nearly 150 years, and today our medicines help more than 51 million people across the globe. Harnessing the power of biotechnology, chemistry and genetic medicine, our scientists are urgently advancing new discoveries to solve some of the world's most significant health challenges, redefining diabetes care, treating obesity and curtailing its most devastating long-term effects, advancing the fight against Alzheimer's disease, providing solutions to some of the most debilitating immune system disorders, and transforming the most difficult-to-treat cancers into manageable diseases. With each step toward a healthier world, we're motivated by one thing: making life better for millions more people. That includes delivering innovative clinical trials that reflect the diversity of our world and working to ensure our medicines are accessible and affordable.

Read More