Ultragenyx Pharmaceutical Inc Announces U.S. FDA Approval of Dojolvi™ For The Treatment of Long-Chain Fatty Acid Oxidation Disorders

Ultragenyx | July 03, 2020

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved Dojolvi™ (triheptanoin) as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD). LC-FAOD are a group of rare, lifelong and life-threatening genetic disorders in which the body is unable to convert long-chain fatty acids into energy. Dojolvi is a highly purified, synthetic, 7-carbon fatty acid triglyceride specifically designed to provide medium-chain, odd-carbon fatty acids as an energy source and metabolite replacement for people with LC-FAOD.

“With today’s FDA approval, patients living with this serious, unpredictable, and often catastrophic disease now have an approved therapy. Many patients with long-chain fatty acid oxidation disorders have difficult lives with frequent hospitalizations and major medical events despite the best current care. Now these patients have an approved treatment as an option to help manage their disease,” said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. “We are grateful to the patients, caregivers, families, dietitians and doctors involved for helping to make this community milestone possible, and we are committed to ensuring that all patients in the U.S. with LC-FAOD who might benefit from Dojolvi will have access to it.”

Spotlight

Data analytics in pharma has altered the way pharmaceuticals were a few years back. In the current era of industry 4.0, big data analytics compelled enterprises to make decisions discriminatively because Artificial Intelligence and Machine Learning prove themselves.

Spotlight

Data analytics in pharma has altered the way pharmaceuticals were a few years back. In the current era of industry 4.0, big data analytics compelled enterprises to make decisions discriminatively because Artificial Intelligence and Machine Learning prove themselves.

Related News

BUSINESS INSIGHTS, PHARMA TECH

Neochromosome Announces Successful Collaboration with Sumitovant Biopharma

Sumitovant Biopharma, Inc. | January 20, 2023

On January 19, 2023, Neochromosome, Inc., announced a partnership with Sumitovant Biopharma, Inc., a technology-driven biopharmaceutical firm expediting the discovery and commercialization of novel therapeutics. This collaboration blends Neochromosome's neoYeast™ platform with Sumitovant's DrugOME™ computational ecosystem to identify new proteins that would benefit from non-canonical amino acid chemistries that are unavailable in nature. As part of the collaboration, Sumitovant used a combination of natural language processing (NLP), artificial intelligence (AI), machine learning (ML), and computational algorithms to identify opportunities for Neochromosome to enhance the discovery and production of proteins limited by evolution and traditional fabrication processes. For example, non-canonical amino acid inclusion allows new-to-nature proteins with improved structures and functions but often requires chemical synthesis, which is expensive, size- and complexity-restricted, and environmentally damaging. neoYeast™, based on Neochromosome's world-leading genome-scale cell engineering technology stack, enables the highly efficient and environmentally friendly integration of non-canonical amino acids into proteins, with both discovery and manufacturing possible in a single genome-engineered yeast strain. Sumitovant used DrugOME to explore libraries of traditional and non-traditional sources of data to recommend targeted protein development candidates for neoYeast. Co-Founder and CEO of Neochromosome, Leslie Mitchell, commented, "The combination of Sumitovant's technology platform and life-sciences expertise with Neochromosome's ability to both discover and biomanufacture novel proteins in a single system enables Neochromosome to accelerate and streamline the delivery of better therapies for patients." She added, "We are excited to partner with the world-class team at Sumitovant to identify opportunities to create better versions of existing drugs, along with new-to-nature compounds, and to manufacture them efficiently at scale." (Source – Business Wire) Sumitovant Biopharma, Inc. The firm has supported the development of multiple FDA-approved products and a strong pipeline of early-through late-stage investigational assets addressing unmet needs in pediatrics, oncology, urology, women's health, specialty respiratory, and infectious diseases through its proprietary computing and data platforms, scientific expertise, and diverse company portfolio. It is a wholly-owned subsidiary of Sumitomo Pharma.

Read More

PHARMACY MARKET, PHARMA TECH

Prescryptive Health Partners with Lilly on First-to-Market Value-Based Program to Stabilize Insulin Pricing

Prescryptive Health | December 16, 2022

Prescryptive Health, a healthcare technology company on a mission to rewrite the script for the U.S. pharmaceutical market, today announced a partnership with Eli Lilly and Company on a new and first-to-market subscription model to help lower costs for people who pay for their Lilly insulin through their employer-sponsored benefits. Through a flat, per-member, per-month subscription fee, employers can offer insulin to their employees with predictable, affordable, and transparent costs. This unique value-based program is designed to create price stability related to insulin utilization, with Lilly refunding a portion of the subscription fee if an employer's total medical spend increases over time for their employees who live with diabetes. "We are proud to partner with Lilly on this unique and first-to-market value-based program. This solution challenges the traditional drug pricing model and reduces the burden on employers who sponsor pharmacy benefits. The result is that it insulates employers against increased drug prices and aligns interests to focus on patient access and health outcomes." Chris Blackley, Prescryptive Health CEO This new program can help address controversial rebates in the pharmacy benefit manager pricing model and will be available through Prescryptive free of any margin or price mark-up, further reducing the cost to employers, health plans, and ultimately healthcare consumers. Additionally, the program offers opportunities for employers to supply insulin at lower costs, or at no cost, to their employees. Added benefits for employers participating in the program include the Prescryptive mobile experience that supports patient engagement and adherence for diabetes care. "In the U.S., more than 34 million people have diabetes, and some people who have employer-based insurance still need affordability solutions to ensure they don't pay too much out-of-pocket for their insulin," said Mike Mason, President, Lilly Diabetes. "At Lilly, we strive to provide new solutions to help lower costs at the pharmacy, including partnering with Prescryptive Health to ensure people with diabetes have access to affordable options to help them reach their treatment goals." According to the American Diabetes Association people with diagnosed diabetes spend about 2.3 times more on medical costs than those without diabetes, which impacts not only every individual patient and plan member, but also employers who sponsor their benefits coverage. Additionally, costs related to the condition include $3.3 billion in increased absenteeism. About Prescryptive Health Prescryptive Health is a healthcare technology company delivering solutions that empower consumers. Prescryptive's secure, mobile-first products connect consumers, pharmacists, and employers, ultimately providing people with the information they need to make informed decisions and take control of their health.

Read More

BUSINESS INSIGHTS, PHARMA TECH

Xtalpi Partners with Singapore's Drug Discovery Platform to Find Better Cancer Treatments

XtalPi Inc. | December 29, 2022

One of the pioneering pharmaceutical technology companies - XtalPi, powered by artificial intelligence (AI) and automation, has recently announced a strategic collaboration with Singapore's national platform for drug discovery and development, the Experimental Drug Development Centre (EDDC), which is hosted by the Agency for Science, Technology, and Research of Singapore (A*STAR). The goal of this partnership is to find novel treatment candidates for non-small cell lung cancer (NSCLC). “According to Research and Markets, the size of the global market for NSCLC treatments will surpass USD 20 billion by 2027” Lung cancer is the second-most prevalent cancer worldwide and the leading cause of cancer-related mortality. NSCLC accounts for more than 80% of all lung cancer occurrences, while its incidence continues to rise worldwide. Despite growing interest and investment in drug research, there is a substantial unmet demand for effective targeted therapeutic alternatives for NSCLC patients to improve outcomes. In this partnership, EDDC and XtalPi will work together to find promising candidates against an NSCLC target chosen by EDDC. XtalPi will use its AI technology, automation platform, and expert domain knowledge. It will also use quantum physics and an AI-driven platform to create and test millions of molecules for the De-novo drug design. Iterations will be used in its robotics-powered synthesis and testing lab to confirm and improve the predicted top-performing molecules with desirable drug properties. EDDC will give helpful information and data about the new NSCLC target, which will speed up the process of finding new drugs and carry the program forward. XtalPi said in a statement that it is delighted to work with EDDC. It intends to help people all over the world by using AI and automation to improve the speed, size, variety, and success rate of drug research and development. In line with this, EDDC stated that it is excited to collaborate with XtalPi to translate science and research into practical and targeted cancer therapies. Furthermore, EDDC is confident that this collaboration will accelerate the development of much-needed targeted therapies for NSCLC patients due to XtalPi's experience in AI and automation technologies. About XtalPi XtalPi is a quantum physics-based, AI-powered drug R&D firm aiming to improve the speed, scale, novelty, and success rate of drug discovery. To meet the needs of its clients and partners, the company has offices in China and the United States. Frost & Sullivan cites the company as one of the world's first AI-powered drug R&D companies, with an iterative feedback loop between dry and wet lab capabilities.

Read More