Circassia gets dual boost for US COPD drugs

PharmaPhorum | April 01, 2019

UK biotech Circassia has had two key decisions go in its favour with FDA, as the company continues with its efforts to rebuild its business after a series of disastrous allergy drug trial failures. However the success means Circassia is looking for ways to pay a series of fees due to AstraZeneca as part of a licensing deal. Circassia gave up on developing a series of drugs for cat, house dust mite, grass, and rag weed allergies after a series of trial flops earlier in the decade. The former darling of UK biotech had raised £200 million with an initial public offering (IPO) to develop the allergy drugs, but switched to a back-up plan by picking up US rights for two unwanted respiratory drugs from AstraZeneca in a deal signed two years ago. Tudorza and Duaklir are inhaled respiratory drugs for chronic obstructive pulmonary disease (COPD) – and at the time of the deal Tudorza (aclidinium bromide), a long-acting muscarinic agonist (LAMA) was already launched in the US and gained approval from the FDA in 2012.

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AskBio Receives EC Orphan Drug Designation for AB-1003 via BrainVectis

Asklepios BioPharmaceutical, Inc. (AskBio) | March 01, 2023

Asklepios BioPharmaceutical, Inc. (AskBio), a subsidiary of Bayer AG, recently announced that the European Commission (EC) has granted orphan drug designation to AB-1003 (also known as LION-101) for the treatment of limb-girdle muscular dystrophy (LGMD). AB-1003 is an investigational recombinant adeno-associated virus (AAV) based gene therapy that is being developed as a one-time intravenous infusion for patients with LGMD type 2I/R9. The disease affects 4.5 people per million worldwide, with more than 5,000 people affected in the EU and the US. The EC decision was received through AskBio's EU-based subsidiary BrainVectis, following a positive opinion from the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) AB-1003 is currently being investigated in a Phase 1/2 multicenter study in the US to evaluate its tolerability, safety, and efficacy in adult subjects with genotypically confirmed LGMD2I/R9. The EC for medicinal products grants orphan drug designation developed to treat a life-threatening disease that affects no more than five people in 10,000 in the EU, provided there is no other adequate treatment option or the medicine can bring significant value to those affected by a specific condition. This designation will provide special incentives in the EU, including eligibility for protocol assistance and possible reductions or exemptions in certain regulatory fees. Additionally, if the medicine is approved for marketing, this designation will provide ten years of marketing exclusivity. Limb-girdle muscular dystrophy (LGMD) is a group of diseases that cause progressive weakening and wasting of the muscles in the arms and legs. The severity, features of LGMD and age of onset vary among the many subtypes of the condition and are often inconsistent, even within the same family. Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) is a form of LGMD caused by mutations in the FKRP gene. Signs and symptoms of LGMD2I/R9 often appear in late childhood and may include difficulty walking and running. The symptoms intensify with time, eventually leading to considerable disability, and afflicted persons typically require a wheelchair for movement roughly 23-26 years after beginning. Unfortunately, there is presently no treatment that slows the advancement of the disease, and management is based on the indications and symptoms that each individual exhibits. About Asklepios BioPharmaceutical, Inc. (AskBio) AskBio is a leading, fully integrated gene therapy company dedicated to developing innovative gene therapeutics and life-saving medicines. With operations worldwide, its pipeline includes gene therapy candidates for various diseases, including the central nervous system, neuromuscular, metabolic, and cardiovascular diseases. AskBio's global headquarters are located in RTP, NC, with European headquarters in Edinburgh. In addition, the company operates AAV research centers in Philadelphia, PA; Columbus, OH; and Paris, and modern clinical and commercial facilities in San Sebastian, Spain.

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BUSINESS INSIGHTS, PHARMA TECH

CONTINUUS Pharmaceuticals Adopts Digital Validation

CONTINUUS Pharmaceuticals | March 15, 2023

On March 14, 2023, ValGenesis, Inc., a leading enterprise validation lifecycle management solutions provider, announced that CONTINUUS Pharmaceuticals has successfully implemented the ValGenesis Validation Lifecycle Management System (VLMS) for efficient management of validation processes across the company in a 100% digital format. CONTINUUS Pharmaceuticals is a rapidly growing small business specializing in end-to-end integrated continuous manufacturing (ICM) of pharmaceuticals. As a spin-out company resulting from a long-term collaboration between MIT and Novartis, CONTINUUS Pharmaceuticals is committed to enhancing the accessibility and affordability of pharmaceutical products worldwide by exploring advanced manufacturing paradigms that expedite the development of raw materials and APIs to final dosage forms. Due to the immense validation requirements involved in the production of different drug products, CONTINUUS Pharmaceuticals needed an electronic solution that determines the disposition of their assets while switching amongst product lines. By leveraging the ValGenesis VLMS, CONTINUUS Pharmaceuticals has successfully implemented facilities, equipment validation, computer system and utility validation. The ValGenesis VLMS is the system of record for CONTINUUS Pharmaceuticals' engineering team and will be expanded to other teams in the future. The highly automated, real-time, and compliant system provided by ValGenesis minimizes the potential for Good Manufacturing Practice (GMP) non-compliance while accelerating time to market. About CONTINUUS Pharmaceuticals CONTINUUS Pharmaceuticals is an end-to-end integrated continuous manufacturing (ICM) company specializing in pharmaceutical production. The company is committed to enhancing the accessibility and affordability of pharmaceutical products worldwide by exploring advanced manufacturing paradigms that expedite the development of APIs and raw materials to final dosage forms. It leverages a novel continuous manufacturing platform called Integrated Continuous Manufacturing (ICM), initially developed through a $65 million joint research project between Novartis and MIT. With ICM, it aims to transform and streamline the supply chain of pharmaceuticals by delivering high-quality, lower-cost drugs "on-demand."

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BUSINESS INSIGHTS, PHARMA TECH

Avita Acquires Q Care Plus, Renames It Avita Care Solutions

Avita Care Solutions | January 10, 2023

PMQ Investors, LLC, the parent company of Avita, has acquired Q Care Plus, Inc. This community-focused care management solution offers stigma-free access to care via telehealth, focusing on telePrEP services. Meanwhile, the terms and conditions of the deal are yet to be revealed. "Bringing Avita and Q Care Plus together comes at a time when reducing health inequities among underserved populations is more critical than ever," said Avita Chief Executive Officer Michael Yount. He further added, "A multiyear pandemic, partisanship-based politics that limit health care rights, and long-standing stigma, fear, and discrimination against marginalized populations like the LGBTQ+ community have exacerbated existing obstacles to health care and created a host of new ones." (Source: PR Newswire) Avita can now broaden its commitment to a community care management strategy that eliminates obstacles for patients and covered entities throughout the care continuum due to its acquisition of Q Care Plus. The company promotes patients' and covered entity partners' capacity to acquire culturally competent LGBTQ+, HIV, PrEP, and sexual wellness care by providing comprehensive access to pharmacy services, 340B program administration, clinical care delivery, and digital health choices. Avita is transitioning to Avita Care Solutions in light of its recent growth. This brand will encompass Avita Pharmaceutical, the company's pharmacy services section, and AvitaCare Atlanta, its new Atlanta medical and primary care center and pharmacy previously owned by AbsoluteCare. There are no plans to rebrand Q Care Plus' digital health platforms. In 2023, Avita will start selling Q Care Plus under the Avita family of brands. About Avita Care Solutions Avita Care Solutions is committed to improving health equity by giving underserved communities comprehensive, caring, and all-inclusive health care and pharmacy services. Its strategy for managing care in the community makes it possible for patients and covered entities to eliminate barriers across the care continuum. Over 145,000 patients and 320+ covered organizations at 65+ locations benefit from Avita's community care management strategy, which includes pharmacy services, program administration, digital health, and clinical care delivery.

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