AZ’s selumetinib for rare disease gets FDA breakthrough status

FDA | April 02, 2019

AstraZeneca and Merck & Co/MSD’s selumetinib has been given a breakthrough designation from the US FDA for a rare paediatric disease, after failing to make the grade in other cancers. The MEK 1/2 inhibitor has picked up the breakthrough status for neurofibromatosis type 1 (NF1), an incurable genetic condition that affects one in 3,000-4,000 newborns worldwide and for which it also received an orphan drug designation from the EMA last year. NF1 can cause lumps under the skin, pigmentation and in around 25% of cases benign tumours on nerve sheaths called plexiform neurofibromas that can cause pain, motor dysfunction and disfigurement. Some patients also experience other complications such as learning difficulties, visual impairment, twisting and curvature of the spine, high blood pressure, and epilepsy. There are no approved therapies for the disease. The new status marks something of a renaissance for selumetinib, which has previously failed as a monotherapy in various solid tumours including thyroid cancer, lung cancer, and uveal melanoma.

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Somalia's first pharmaceutical factory since the start of the country's civil war has opened its doors in Mogadishu. The Horn of Africa nation has not had such a firm operating there since the breakout of violence in the country three decades ago.

Spotlight

Somalia's first pharmaceutical factory since the start of the country's civil war has opened its doors in Mogadishu. The Horn of Africa nation has not had such a firm operating there since the breakout of violence in the country three decades ago.

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BUSINESS INSIGHTS, PHARMA TECH

ClinChoice Announces Acquisition of CROMSOURCE

ClinChoice | March 03, 2023

ClinChoice, a leading global clinical CRO, recently announced that it has acquired CROMSOURCE S.r.l., an ISO-certified full-service contract research organization. CROMSOURCE has its corporate headquarters in Verona, Italy, its US headquarters in Cary, NC, and subsidiaries in the US and seven countries throughout eastern and western Europe. With a proven track record of providing excellent clinical research and staffing services to multinational pharmaceutical, biotechnology, and medical device clients, CROMSOURCE's acquisition will strengthen ClinChoice's global network and further enhance its full-service capabilities. ClinChoice is known for its commitment to providing high-quality services and timely delivery, resulting in the successful completion of clinical trials and functions, including regulatory, biometrics, safety, medical, and clinical operations across various sectors such as drug, medical devices, vaccines, cosmetics, and consumer health using both project-based and FSP models. The company offers services covering the development lifecycle for biotechnology, pharmaceutical, consumer products and medical device companies worldwide. With a broad clientele ranging from the largest multinationals to startup and emerging companies, ClinChoice has completed over 1,500 clinical studies in various therapeutic areas such as CNS, oncology, ophthalmology, rare disease, respiratory and metabolic. CROMSOURCE's acquisition was advised by Fairmount Partners, while Crosstree Capital represented ClinChoice. About ClinChoice ClinChoice is a leading global clinical CRO that provides comprehensive services to biotechnology, pharmaceutical, medical device, and consumer products clients worldwide. With a successful 25-year track record, it is considered the standard for excellence in clinical operations, biostatistics, data management, CDISC, statistical programming, regulatory affairs, medical affairs, and pharmacovigilance. The company has established major delivery centers in Europe, the US, Canada, China, India, Japan, and the Philippines, employing over 3,000 people worldwide. ClinChoice has a strong and talented team and is expanding its clinical operations in seven countries across North America, Asia, and Europe.

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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.

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BUSINESS INSIGHTS, PHARMACY MARKET

Harrow Introduces Next-Gen Compounded Atropine Formulations

Harrow | February 16, 2023

On February 15, 2023, Harrow, a renowned pharmaceutical firm specializing in eyecare in the United States, announced the launch of its next-generation, patent-pending compounded Atropine formulations. These formulations are now accessible through ImprimisRx, a mail-order pharmacy subsidiary wholly owned by Harrow. The following are the key features of ImprimisRx's next-generation Atropine formulations Thoroughly validated analytical tests are conducted to ensure potency, consistency, and stability. The formulations are free of preservatives and boric acid. They possess a physiologic pH of 5.5. They can be stored at room temperature for up to 180 days. Eyecare professionals can purchase these compounded Atropine formulations for as less as $39 per 5mL bottle through various channels, such as online via atropine.com or by contacting ImprimisRx Customer Service at 615-437-4690 via call or text. Compounded formulations are recommended for patients with clinical requirements that are not met by FDA-approved medications. It is essential to note that compounded medications are not reviewed by FDA for their safety and effectiveness. ImprimisRx does not manufacture copies of products that are already available in the market. Harrow's Chairman and Chief Executive Officer, Mark L. Baum, commented, "Our mission has always been to help our eyecare professionals address their patients' unmet needs and to make ophthalmic medications both accessible and affordable. The availability of these innovative compounded atropine formulations is in keeping with our mission. We believe ophthalmologists, optometrists, and the millions of Americans who may benefit from topical atropine therapy, will appreciate the tremendous value these patent-pending formulations offer." (Source – Business Wire) About Harrow Founded in 2012, Harrow is a leading ophthalmic pharmaceutical firm in the United States focused on discovering, developing, and introducing accessible, innovative, and affordable prescription therapies. It holds commercial rights for ten FDA-approved eyecare pharmaceutical products. In addition, the company also operates ImprimisRx, a leading U.S. compounding business that specializes in ophthalmic medications and is licensed to ship prescription drugs in all 50 states. The success of its efforts is reflected in its remarkable financial performance, with a 72% compound annual growth rate achieved during our first eight years in operation. This phenomenal growth rate has established Harrow as one of the fastest-growing ophthalmic pharmaceutical companies in the United States.

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