FDA grants breakthrough designation to Achillion’s PNH drug

Pharmaceutical Technology | September 26, 2019

The US Food and Drug Administration (FDA) has granted Achillion Pharmaceuticals breakthrough designation for danicopan (ACH-4471) for patients with paroxysmal nocturnal haemoglobinuria (PNH). Achillion is studying danicopan combined with a C5 monoclonal antibody in a Phase II trial of patients with a suboptimal response to C5 inhibitors alone, the standard of care for the condition. The FDA’s designation is based upon positive efficacy and safety data from this ongoing study. PNH is a rare, genetic blood disease caused by somatic mutation, which results in the absence of key receptors, CD55 and CD59, on the surface of red blood cells. These are recognised by the alternative pathway (AP) as foreign and destroyed in the blood stream and in the liver. CD5 inhibitors targets intravascular haemolysis where these red blood cells are attacked in the blood stream, but, according to Achillion, 75% of those treated with C5 alone remain anaemic during treatment with a third needing blood transfusions.

Spotlight

Precision medicine, sometimes called personalized medicine, is an approach for protecting health and treating disease that takes into account a person’s genes, behaviors, and environment. Interventions are tailored to individuals or groups, rather than using a one-size-fits-all approach in which everyone receives the same care.

Spotlight

Precision medicine, sometimes called personalized medicine, is an approach for protecting health and treating disease that takes into account a person’s genes, behaviors, and environment. Interventions are tailored to individuals or groups, rather than using a one-size-fits-all approach in which everyone receives the same care.

Related News

PHARMA TECH

Hovione and Zerion Pharma announce a strategic partnership to market the Dispersome® technology platform

Hovione | February 23, 2022

Hovione, the leader in spray drying and particle engineering, announced a strategic partnership with Zerion Pharma to market and commercialize Dispersome®, Zerion´s proprietary solubility enhancement technology platform. Zerion´s innovative Dispersome® technology builds on a new concept of increasing drug solubility by using natural protein-based excipients to formulate APIs into amorphous solid dispersions (ASDs) by spray drying. By combining Dispersome® technology with Hovione's unique spray drying capabilities, experience in development, scale-up and GMP manufacturing, Hovione further strengthens its leadership in amorphous solid dispersions and shows its commitment to innovative solutions to overcome one of the most prevalent challenges faced by the industry: low drug solubility. "We are extremely pleased to have entered into this partnership with Zerion whom we recognize as a very innovative company in the field of oral drug formulations. By joining forces, Zerion and Hovione will be able to bring Dispersome® to the market faster. We look forward to applying the technology in collaboration with our customers and partners in our effort to develop novel drugs with strong benefits to patients. Hovione will continue to pursue opportunities to enhance its offering around core areas of expertise such as particle engineering and inhalation by partnering with companies and research institutions developing innovative drug formulation technologies, like Dispersome®". Jean-Luc Herbeaux, Hovione´s Chief Operating Officer Zerion´s CEO, Ole Wiborg, sees a strong synergy in the partnership and expects it to generate new pharma partnerships. "We consider Hovione the global leader in the field of producing amorphous solid dispersions by spray-drying. More importantly for us, this leadership has resulted in Hovione being, over the last 5 years, the main commercial manufacturer of novel FDA-approved drugs formulated as amorphous solid dispersions. Incorporating these competencies in our offering to the pharma industry both validates the strength of our Dispersome® platform and provides us and our pharma partners immediate access to the highest quality in upscaling and commercial GMP manufacturing." says Ole Wiborg. The announcement of this partnership follows Hovione's communication on a further expansion with an expected investment of $170 million in assets worldwide and shows the company's commitment to also expand its technology platforms. Hovione is investing both in new assets and innovative technologies to meet customer demand for integrated and differentiated services in drug substance manufacturing, particle engineering and most recently drug product manufacturing. About Hovione Hovione is an international company with over 60 years of experience as a Contract Development and Manufacturing Organization (CDMO) with a fully integrated offering of services for drug substance, drug product intermediate and drug product. The company has four FDA inspected sites in the USA, Portugal, Ireland and China and development laboratories in Lisbon, Portugal and New Jersey, USA. Hovione provides pharmaceutical customers services for the development and compliant manufacture of innovative drugs, including highly potent compounds, and customized product solution across the entire drug life cycle. In the inhalation area, Hovione is the only independent company offering a complete range of services, from API, formulation development and devices. Hovione is a company with a culture based on innovation, quality and delivery. Hovione was the first Chemical/ Pharmaceutical Company to become a Certified B Corp, is a member of Rx-360, EFCG and participates actively in industry quality improvement initiatives to lead new global industry standards. About Zerion Pharma ApS Zerion has pioneered the Dispersome® technology that greatly enhances the solubility of poorly soluble, oral drugs and improves bioavailability and therapeutic outcomes for the patients. The Dispersome® technology is based on preparing stable amorphous formulations by mixing high loads of the drug compound with beta-lactoglobulin, a sustainable and biodegradable by-product from cheese production. Zerion develops proprietary drug formulations and offers the Dispersome® technology to established pharma companies as a means to solve their most challenging drug solubility problems.

Read More

Additive Orthopaedics® Announces additive manufacturing Capability with Patient Specific Implant Locking Technology

Additive Orthopaedics, LLC | October 23, 2020

Additive Orthopaedics, LLC., the leader in 3D printed orthopaedic foot and ankle devices, announces a novel breakthrough in orthopaedic additive manufacturing with their ability to incorporate locking technology in all of their Patient Specific Implants. The patent pending technology can be incorporated in Additive Orthopaedics Patient Specific 3D Printed Plates and Patient Specific 3D Printed Bone Segments. According to Luciano Bertolotti, Director of Engineering for Additive Orthopaedics, "Locking technology is straightforward for off the shelf implants in which the machining process for the screw hole threads is set up once and can be manufactured in high volumes.

Read More

PHARMA TECH

KSP Specialty Pharmacy now offers access to non-oncology drugs and launches new website featuring pharmacy patient portal

Karmanos Cancer Institute | October 05, 2021

The Barbara Ann Karmanos Cancer Institute and KSP Specialty Pharmacy (KSP) are pleased to announce the launch of new services and a new website: www.ksppharmacy.org. The new site features provider resources and information for payers and pharmaceutical companies. Most notably, the site includes an online portal where patients can refill their prescriptions. This tool also allows patients to see their list of medications, get reminders and interact with their pharmacist. A portal for patient education and tips is forthcoming. Additionally, KSP has begun offering non-oncology specialty drugs to serve providers and patients throughout the McLaren Health Care network. KSP is a specialty pharmacy within the McLaren Health Care Network, serving the needs of patients with complex disease states and high-cost therapeutic treatments. KSP began as an extension of the Barbara Ann Karmanos Cancer Institute in 2019 and has since grown to assist with specialty medication needs of patients throughout the McLaren system in Michigan, Ohio and Indiana. Additionally, the Karmanos ambulatory pharmacy, which provides non-specialty medication, has launched a portal with similar functionality. Those undergoing cancer treatment at Karmanos can now access their prescription information and request refills on the Karmanos pharmacy web page. We are continually striving to provide our patients with tools and convenience to help them understand their treatment. The new website and app will provide the outstanding service we offer over the phone and in person on a new, convenient platform, - Stephen Smith, RPh, MS, FASHP, Chief Pharmacy Officer. KSP provides patient care and customer service through a panel of expert clinical pharmacy specialists that can assist in drug education and management of not only a patient's specialty prescriptions, but their entire prescription portfolio. The pharmacy team has direct access to the patient's medical records and providers to ensure the time to delivery of the first dose is swift. In fact, the average rate to the first dose at KSP during fiscal year 2021 is 3.92 days, while the industry standard is more than ten days. A primary goal is to ensure that our in-house social worker and account resolution team find unique programs to allow patients access to high-cost medications without being financially burdened. This allows patients to stay adherent and compliant with their prescribed therapies and start a path to faster healing and recovery. Specialty medications are used to treat complex health conditions that include hepatitis C, multiple sclerosis, cancer, rheumatoid arthritis, organ transplant, migraines and others. KSP Specialty Pharmacy's expanded services include the ability to dispense medications for patients within the McLaren Health System who face these disease states. These medications may need to be administered by a health care professional, self-injected or taken by mouth, which may require clinical monitoring. Specialty medications often require special handling, delivery and storage requirements. KSP is accredited by the Utilization Review Accreditation Commission (URAC) and the Accreditation Commission for Health Care (ACHC). URAC designation was earned in 2020 and allows for the provision of agents in other specialized areas, such as HIV, gastroenterology, neurology and many others. About the Barbara Ann Karmanos Cancer Institute Karmanos Cancer Institute is a leader in transformative cancer care, research and education through courage, commitment and compassion. Our vision is a world free of cancer. As part of McLaren Health Care, Karmanos is the largest provider of cancer care and research in the state of Michigan. For more than 75 years, our administrative and research headquarters, along with our premier specialty cancer hospital, have been located in downtown Detroit. Our 15 network sites throughout the state deliver market-leading cancer care and clinical trials conveniently to the communities where many of our patients live. Karmanos is recognized by the National Cancer Institute as one of the best cancer centers in the nation. Our academic partnership with the Wayne State University School of Medicine provides the framework for cancer research and education – defining new standards of care and improving survivorship.

Read More