ICER deems unapproved Aimmune, DBV peanut allergy drugs unaffordable, sparking backlash

fiercepharma | July 12, 2019

Two small companies, Aimmune Therapeutics and DBV Technologies, are racing to break into what analysts expect will be a billion-dollar market for drugs to treat peanut allergies. But neither of their experimental treatments, Aimmune’s AR101 and DBV’s Viaskin, has been approved by the FDA, nor have the companies said anything about pricing plans. That didn’t stop the Institute for Clinical and Economic Review (ICER) from issuing a dreaded “affordability alert” on the drugs, however. The report, released earlier this week, blasted both drugs for being unaffordable based on analysts’ estimates that AR101 could cost $4,200 a year and Viaskin $6,500. AR101 and Viaskin were designed to build an immune tolerance to peanuts in children with the allergy. With the help of an independent appraisal committee, ICER determined that there wasn’t enough evidence to prove that either drug offers a health benefit over simply avoiding peanuts. In order to avoid crossing ICER’s acceptable annual healthcare budget threshold of $819 million, Aimmune would need to capture 41% of eligible patients and DBV 71%, ICER concluded. David Rind, M.D., ICER’s chief medical officer, acknowledged in a statement that the drugs have been proven to desensitize patients to the peanut allergy, but that they also raise the risk of “increased allergic reactions” and use of epinephrine—the shot kids and parents carry around to stop life-threatening reactions. “Caregivers and patients must remain vigilant about avoiding peanuts even while on desensitization therapy, and future clinical trials will need to demonstrate whether long-term benefits of these approaches outweigh their short-term risks,” Rind said.

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This white paper reviews how the Process Analytical Technologies (PAT) originally developed by the FDA for the pharmaceutical industry can also improve quality and efficiency in medical device manufacturing. The underlying principle of the PAT approach is that quality should be designed into the product based on a fundamental understanding of the critical manufacturing processes.


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Cambrex to Acquire Snapdragon Chemistry, a Leader in Continuous Flow API Development Services

Cambrex | November 22, 2022

Cambrex, a leading global contract development and manufacturing organization providing drug substance, drug product, and analytical services across the entire drug lifecycle, today announced that it has entered into a definitive agreement to acquire Snapdragon Chemistry, a leading US-based provider of chemical process development services to a broad range of emerging and established biopharma customers. Snapdragon specializes in active pharmaceutical ingredient batch and continuous flow process development, utilizing state-of-the-art automation technology and proprietary equipment to solve complex process and analytical development challenges. The team of scientists and engineers apply deep process understanding afforded by data-rich experimentation to design and rapidly execute efficient GMP and non-GMP manufacturing processes. With R&D and manufacturing headquartered in Waltham, Massachusetts, Snapdragon's 74 employees come with strong ties to the local scientific community, with 31 PhD scientists on staff. "The acquisition of Snapdragon will accelerate our growth in the area of continuous flow process development and manufacturing, complementing our recent organic investments in our High Point, North Carolina facility. With R&D and manufacturing capabilities in the heart of Boston's biopharma hub, Snapdragon will continue to focus on solving their customers' most difficult process development challenges." Tom Loewald, CEO of Cambrex "We are excited to be joining Cambrex, a company with over 40 years of drug substance development and manufacturing expertise," said Matt Bio, CEO of Snapdragon. "Partnering our best-in-class process development capabilities with Cambrex's larger scale manufacturing facilities in North America and Europe is a natural fit, both for our employees and our customers." Snapdragon recently opened its second facility, a new 51,000-square-foot facility to manufacture experimental pharmaceutical products for human clinical trials. The new facility expanded the company's capacity for supplying clinical intermediates and drug substances. The transaction is expected to close following the completion of customary regulatory approvals. This will be Cambrex's second acquisition within a year along with Q1 Scientific, consistent with its strategy to expand its portfolio of specialized solutions for pharmaceutical development and manufacturing. About Cambrex Cambrex is a leading global contract development and manufacturing organization that provides drug substance, drug product, and analytical services across the entire drug lifecycle. With over 40 years of experience and a growing team of over 2,300 experts servicing global clients from North America and Europe, Cambrex is a trusted partner in branded and generic markets for API and finished dosage form development and manufacturing. Cambrex offers a range of specialized drug substance technologies and capabilities, including biocatalysis, continuous flow, controlled substances, solid-state science, material characterization, and highly potent APIs. In addition, Cambrex can support conventional dosage forms, including oral solids, semi-solids, and liquids, and has the expertise to manufacture specialty dosage forms such as modified-release, fixed-dose combination, pediatric, bi-layer tablets, stick packs, topicals, controlled substances, sterile, and non-sterile ointments.

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Everest Medicines Appoints Rogers Yongqing Luo as Chief Executive Officer to Lead Next Stage of Company Growth

Everest Medicines | September 19, 2022

Everest Medicines a biopharmaceutical company focused on developing and commercializing transformative pharmaceutical products to address critical unmet needs in Asia Pacific markets, announced that it has appointed Rogers Yongqing Luo as Chief Executive Officer of the Company, effective immediately. Mr. Luo will also serve as an Executive Director on Everest's Board of Directors. Mr. Luo brings to Everest more than 25 years of experience in the healthcare industry with an extensive leadership track record across clinical development, regulatory affairs and commercialization. Previously, Mr. Luo served as President and General Manager, Greater China at Brii Biosciences and CEO of TSB Therapeutics where he spearheaded the rapid registration approval, GMP certification and successful commercialization of the amubarvimab/romlusevimab combination, the first COVID-19 neutralizing antibody treatment in China. Prior to that, he was Global Vice President and China General Manager at Gilead Sciences where he led the clinical development, regulatory approval and successful commercial launch of eight innovative therapeutics, and efforts that rapidly expanded patient access across China. Mr. Luo previously oversaw the successful launch of several high-profile medicines. He managed sales, marketing, government affairs and market access functions for various leading multinational pharmaceutical companies including Roche and Novartis, and worked two years at Novartis headquarter in Switzerland. "As Everest Medicines continues to evolve our business strategy and expand our novel product pipeline at a rapid pace, we are thrilled to have Rogers join the Company to lead Everest through its next phase of growth in becoming a fully-integrated biopharma company in China. With his robust background as a top executive at both biotech startup and large multinational pharma companies, Rogers brings to Everest a unique set of expertise including comprehensive scientific insight, proven outstanding leadership and excellent commercialization capabilities in patient access, and a demonstrated history of successfully advancing early- and late-stage innovative pipeline to commercial launch in Greater China. In particular, Rogers' leadership will strengthen Everest's new product development and commercial capabilities as we progress toward potential approvals of our late-stage anchor product candidates, including Nefecon, Xerava, etrasimod and mRNA vaccines. We look forward to leveraging Rogers' leadership and industry expertise to generate long-term growth and success across the business." Mr. Wei Fu, Chairman of Everest Medicines and Chief Executive Officer of CBC Group "I'm very excited to join Everest and I look forward to working with this talented, world-class team of scientists and industry professionals to further the Company's industry leadership. We will continue to advance our late-stage pipeline towards commercialization and with our strong balance sheet, we aim to execute synergistic business development deals to accelerate our path to become an integrated biopharma," said Rogers Yongqing Luo, Chief Executive Officer of Everest Medicines. "We will continue to invest into our discovery platform to build an innovative therapeutic pipeline for the benefit of patients with unmet needs in Greater China and other Asia Pacific markets, and to create sustained, long-term value for our shareholders." About Everest Medicines Everest Medicines is a biopharmaceutical company focused on developing and commercializing transformative pharmaceutical products that address critical unmet medical needs for patients in Asian markets. The management team of Everest Medicines has deep expertise and an extensive track record of high-quality clinical development, regulatory affairs, CMC, business development and operations both in China and with leading global pharmaceutical companies. Everest Medicines has built a portfolio of potentially global first-in-class or best-in-class molecules, many of which are in late-stage clinical development. The Company's therapeutic areas of interest include oncology, autoimmune disorders, cardio-renal diseases and infectious diseases.

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Kubota Vision Announces Collaboration Agreement with Leading Diabetes Center

PBOS | September 30, 2022

Kubota Vision Inc. a clinical-stage specialty ophthalmology company and a wholly-owned subsidiary of Kubota Pharmaceutical Holdings Co., Ltd. announced today that the Company and Joslin Diabetes Center have entered into a material transfer and collaboration agreement for our Patient Based Ophthalmology Suite in-home optical coherence tomography device. Joslin Diabetes Center is conducting two clinical studies to evaluate the ability of PBOS to identify cases of diabetic macular edema that may need treatment compared to a commercially-available OCT device. The studies will be led by Dr. Paolo S. Silva at Joslin Diabetes Center. Dr. Silva is the Co-Chief of Telemedicine at the Beetham Eye Institute of the Joslin Diabetes Center. His work is focused on innovative and investigative work in a field at the intersection of clinical care and technology with the hope of providing an ideal model for the delivery of evidence-based, highly effective, and efficient diabetes eye care to the population that needs it the most. Joslin Diabetes Center, which is affiliated with Harvard Medical School, is a one-of-a-kind institution on the front lines of the world epidemic of diabetes — leading the battle to conquer diabetes in all of its forms through cutting-edge research and innovative approaches to clinical care and education. Dr. Silva stated, “Through these studies, we will determine if home-based in-home optical coherence tomography is useful in the evaluation of diabetic macular edema and how we monitor patients at risk for losing vision from the condition.” “Diabetic retinopathy is one of the most common diseases in the world. The use of our technology brings us great excitement as we continue to focus on the development of medical devices and explore further possibilities.” Ryo Kubota, MD, PhD, Chairman, President, and CEO of Kubota Vision Inc. About PBOS PBOS is a low cost, home-based, ophthalmic self-monitoring OCT device. This small handheld device addresses needs in mobile Health applications for self-monitoring of retina health by patients, in the home and in remote field locations. The PBOS aims to improve ophthalmic treatment outcomes in patients diagnosed with and treated for wet age-related macular degeneration, diabetic macular edema and other neovascular retinal diseases. PBOS is being designed to detect nascent disease progression and support patient re-treatment prior to irreversible vision loss due to disease progression. Key features are low cost and a patient-friendly design, to be used directly by patients at home. PBOS is being designed to capture changes in retinal anatomy. Network connectivity and cloud-based technologies are used to alert the patients and their physicians of disease progression and re-treatment needs. About Kubota Vision Inc. Kubota Vision Inc. is a wholly owned subsidiary of Kubota Pharmaceutical Holdings Co., Ltd. committed to translating innovation into a diverse portfolio of drugs and devices to preserve and restore vision for millions of people worldwide. Kubota Pharmaceutical group’s pipeline includes a wearable device for myopia control using Kubota GlassTM technology and a handheld OCT device for the monitoring of neovascular retinal diseases, to be used directly by patients.

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Aquestive Therapeutics Receives FDA Tentative Approval for Libervant™ (diazepam) Buccal Film

Aquestive Therapeutics, Inc. | August 31, 2022

Aquestive Therapeutics, Inc. a pharmaceutical company advancing medicines to solve patients' problems with current standards of care and provide transformative products to improve their lives, announced today that the U.S. Food & Drug Administration has granted tentative approval for Libervant™ Buccal Film for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity that are distinct from a patient’s usual seizure pattern in patients with epilepsy 12 years of age and older. "Tentative approval" means the FDA has concluded that Libervant has met all required quality, safety, and efficacy standards for approval but, due to an existing FDA regulatory grant of orphan drug market exclusivity for Valtoco®, a diazepam nasal spray product, Libervant is not yet eligible for marketing in the United States. As a result of the FDA determination, the Agency cannot give final approval for Libervant until the expiration or inapplicability of the orphan drug market exclusivity, including, for example, by court order, a selective waiver of the orphan drug exclusivity, or a reversal of the FDA’s decision and determination that Libervant is “clinically superior” to Valtoco. “The FDA’s decision provides welcome clarity to our business. The tentative approval of Libervant is a significant achievement that brings us one step closer to bringing this important medicine to patients who are unable or choose not to use the current standards of care. Libervant has the potential to offer these patients a treatment option that is simple, portable, and precise. A significant unmet need exists for additional alternate delivery options given the ongoing shortage of diazepam rectal gel, which continues to represent a substantial portion of the current diazepam rescue market. This FDA action further validates our ability to gain FDA approval of our pipeline programs. We look forward to continuing the rapid progression of AQST-109 for the treatment of severe allergic reactions, including anaphylaxis, towards a filing with the FDA.” Daniel Barber, Chief Executive Officer of Aquestive “We continue to examine the FDA’s determination on Libervant market access and plan to request a meeting with the Agency as soon as possible. We disagree with the FDA’s determination on the various points presented by the Agency and believe that, particularly in the case of our submitted studies on the effect of food on the absorption of diazepam formulations, Libervant has the distinct advantage of being able to be readily administered when needed without regard to food, providing an important benefit to patients. This is important to patients as rapid and extensive drug absorption is critical in a rescue situation. We will continue to vigorously engage with the FDA on the need for these data to be carefully considered.” During the review process, the Company submitted to the FDA the results of a 2021 Aquestive sponsored randomized, open-label, two-sequence, two-period, two-treatment crossover study to evaluate the effect of food on the pharmacokinetics of Valtoco in healthy adult subjects. The results of this study indicated that, when Valtoco is administered after a high fat meal, the maximum drug concentration (Cmax) was reduced by 48% compared to Valtoco administered to subjects in a fasted state. The study also showed that the time to maximum drug concentration (Tmax) of Valtoco doubled from 2 hours to 4 hours when administered after a high fat meal. Aquestive provided the data along with a cross-study comparison to a similar study performed with Libervant, to FDA during the review process. The FDA’s decision concluded that the information Aquestive submitted was not sufficient to overturn the Agency's previous conclusion regarding the lack of food effect for Valtoco. Specifically, the FDA stated that “[a] cross-study comparison is not considered [by the reviewer] to be a suitable approach for making a quantitative comparison of plasma concentrations between different products because of the lack of a common reference standard between the two studies.” Aquestive will seek to gain alignment with the Agency on a reasonable path to appropriately characterize the food effect of Valtoco including potentially conducting a comparative study as indicated by the FDA. About Libervant Libervant™ is a buccally, or inside of the cheek, administered film formulation of diazepam, a benzodiazepine intended for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity that are distinct from a patient’s usual seizure pattern in patients with epilepsy 12 years of age and older. Aquestive developed Libervant as an alternative to the device-based products currently available for patients with refractory epilepsy, including a rectal gel and nasal spray products. Approximately 1.0 million patients with epilepsy suffer from uncontrolled refractory seizures, approximately 85% of whom will not interact with the available treatments. About Aquestive Therapeutics Aquestive Therapeutics, Inc. is a pharmaceutical company advancing medicines to solve patients’ problems with current standards of care and provide transformative products to improve their lives. We are developing orally administered products to deliver complex molecules, providing novel alternatives to invasive and inconvenient standard of care therapies. Aquestive has five commercialized products on the U.S. market, four licensed products and one stand-alone proprietary product to date, Sympazan® (clobazam) oral film for the treatment of seizures associated with Lennox-Gastaut syndrome. Our licensees market their products in the U.S. and around the world. The Company also collaborates with pharmaceutical companies to bring new molecules to market using proprietary, best-in-class technologies, like PharmFilm®, and has proven drug development and commercialization capabilities. Aquestive is advancing a late-stage proprietary product pipeline focused on treating diseases of the central nervous system, or CNS, and an earlier stage pipeline for the treatment of severe allergic reactions, including anaphylaxis.

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Spotlight

This white paper reviews how the Process Analytical Technologies (PAT) originally developed by the FDA for the pharmaceutical industry can also improve quality and efficiency in medical device manufacturing. The underlying principle of the PAT approach is that quality should be designed into the product based on a fundamental understanding of the critical manufacturing processes.

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