PANTHERx® Rare Pharmacy Selected by Alnylam Pharmaceuticals to Distribute GIVLAARI™ (givosiran)

PR Newswire | December 12, 2019

PANTHERx® Rare Pharmacy Selected by Alnylam Pharmaceuticals to Distribute GIVLAARI™ (givosiran)
PANTHERx® Rare Pharmacy announces that it has been selected by Alnylam Pharmaceuticals as a very limited distribution partner for the novel drug GIVLAARI™ (givosiran). GIVLAARI is the first and only FDA-approved medicine indicated for the treatment of adults with Acute Hepatic Porphyria (AHP). GIVLAARI becomes the second RNAi therapeutic from Alnylam approved by the FDA in the last 16 months and the first-ever approved GalNAc-Conjugate RNA therapeutic, a landmark in the advancement of precision genetic medicines. The FDA approval of GIVLAARI was received less than four months after the New Drug Application (NDA) filing acceptance and was based on positive results from the ENVISION Phase 3 study, a randomized, double-blind, placebo-controlled,

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PHARMA TECH

Invitae to Acquire Ciitizen to Strengthen its Patient-Consented Health Data Platform to Improve Personal Outcomes and Global Research

Invitae | September 07, 2021

Invitae , a leading medical genetics company, today announced it has entered into a definitive agreement to acquire Ciitizen, a patient-centric consumer health tech company. Backed by a16z, Section 32 and Verily, Ciitizen is working to build a global platform to help patients collect, organize, store and share their medical records digitally. The acquisition would enhance Invitae's platform by providing patients an easy-to-use, centralized hub for their genomic and clinical information, which together comprise a powerful dataset with the potential to drive research and improve healthcare decision making. "Invitae is relentlessly pursuing a day when genetics is used routinely to improve healthcare for billions of people around the globe. We believe combining Ciitizen's state-of-the-art, transparent and patient-consented platform with our technologies and services will accelerate our evolution into a genome information company that informs healthcare throughout one's life. This would give us the ability to engage patients, create an innovative patient-centered data ecosystem and deliver better outcomes for everyone. Invitae views the acquisition of Ciitizen as an important part of its strategy to be the industry leader across the genetic testing, software and health information technology spaces," said Sean George, Ph.D., co-founder and CEO of Invitae. "Ciitizen has already helped patients take ownership of their medical records to optimize therapy for themselves and contribute their data towards a variety of goals, including research on rare diseases," said Anil Sethi, CEO and founder of Ciitizen. "Patients leave a breadcrumb trail of records behind them, and we believe access to this real-world data is key to personalized therapeutics in the future. The combination of lifelong health history together with Invitae's world-class genetic and data services would enable a digital ecosystem of personalized services for each patient. Democratization of patient-consented data is expected to improve outcomes by providing access to third-parties for whom the patient has granted consent. Unlike many health data companies, we operate on the premise that it's the patient's data, not ours." Invitae is working to aggregate results from the world's genetic tests into a single, easy-to-use service that makes genetic information accessible to all who can benefit from it - always with patient consent. Over the past five years, Invitae has completed 13 acquisitions that have expanded its testing menu, lowered the cost of its tests, improved its customer experience and enhanced its ability to attract partners. Invitae raised more than $1.4 billion since the beginning of 2021, in part to identify and acquire key assets that bring the company closer to delivering on its vision. The addition of Ciitizen is expected to empower Invitae to more quickly extend its platform, adding a complementary patient service that streamlines the process of collecting and organizing health information from any source and uses machine learning to transform the unstructured (and largely untapped) health information to enable better care decisions, find clinical trials, and power research using real-world evidence. In addition, as the use of Invitae testing and the Ciitizen service expands, third-party access to consented genomic and other clinical data can be democratized to power a variety of applications that can enhance Invitae's business and growth strategy. About Invitae Invitae Corporation is a leading medical genetics company, whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. About Ciitizen Ciitizen is a patient-centric consumer health technology platform that enables patients to collect, store and share their medical records in a single location. Ciitizen developed proprietary machine learning and AI technologies to abstract critical information from unstructured health records to advance the understanding of diseases, match patients with potentially life-saving clinical trials, and enable better care decisions. Ciitizen also provides valuable big data analytics and data quality technologies to the network of Health Information Exchanges (HIEs) to produce research-ready, regulatory-grade data within minutes, instead of hours or days, and ensure HIEs can comply with the Information Blocking regulations.

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Seven pharmaceutical stocks that stand to win beyond the Covid-19 chase

InvestorPlace | July 15, 2020

It’s easy to miss the forest for the trees in markets like this. Especially when it comes to pharmaceutical stocks. What I mean is that, in some sectors, there are great stocks that get overlooked because a few of them are working on event-driven issues, while others are creating long-term solutions to major trends. This is especially true in the pharmaceutical and biotech spaces right now. Maybe it’s because the Kentucky Derby was postponed, but people are betting on pharmas discovering a vaccine to Covid-19 like it’s the only horse race out there. The fact is, there’s a whole racing season and betting on one horse in one race isn’t successful investing. And neither is betting on pharmaceutical stocks for one medicine. That’s where my grading system comes in. Overall, there are plenty of massively successful drugs. However, they’re blockbusters because they can be used for a variety ailments. Or, the top companies have a strong pipeline of drugs that address issues that are important to a large number of people.

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PHARMACY MARKET

Avacta Announces FDA Approval of its Investigational New Drug (IND) Application for AVA6000

Avacta | November 29, 2021

Avacta Group plc, a clinical stage biopharmaceutical company developing innovative cancer therapies and powerful diagnostics based on its proprietary Affimer® and pre|CISION™ platforms, is pleased to announce that the US Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application for AVA6000. This will allow the Group to expand its Phase I clinical trial, ALS-6000-101, into clinical trial sites in the United States. AVA6000 is a novel form of doxorubicin that has been modified with Avacta’s pre|CISION™ platform to improve its safety and therapeutic index. Anthracyclines such as doxorubicin, a generic chemotherapy for which the market is expected to grow to $1.38bn by 20241, are widely used as part of standard of care in several tumour types, but their use is limited by cumulative toxicity. AVA6000 has been designed to limit cell penetration of the drug, and therefore its cell killing effect, until it is specifically activated by fibroblast activation protein α (FAP) which is in high concentration in many solid tumours compared with healthy tissues. The resulting reduced exposure of healthy tissues to active doxorubicin has the potential to significantly increase its therapeutic index by reducing the incidence of adverse effects, including cardiotoxicity and myelosuppression. The FDA has completed its 30-day review of Avacta’s IND application, which was submitted ahead of schedule in October 2021, and has concluded that the Group may proceed with its proposed clinical investigation. This allows Avacta to enroll eligible patients into US clinical trial sites for the company’s Phase I multi-centre study, ALS-6000-101. As previously announced in August 2021, the Company has begun recruiting and dosing patients for this study at several clinical trial sites in the UK, and continues to expect the dose escalation phase for this trial to complete by Q2 2022 followed by completion of the dose expansion phase around mid-2023. Enrollment in US clinical trial sites is expected to begin in early 2022. “We are delighted to have received approval from the FDA to add clinical trial sites in the United States as part of the Phase I study for AVA6000. This is a major milestone in our development of pre|CISION™ chemotherapies and is testament to the performance of our clinical development team and the quality of the pre-clinical data for AVA6000. Dr. Alastair Smith, Chief Executive of Avacta Group Provided that the study shows that the pre|CISION™ technology is effective in reducing systemic toxicity of Doxorubicin, then that would open up an extensive and proprietary pipeline for Avacta of next-generation pre|CISION™ chemotherapies with significant clinical and commercial advantages in a chemotherapy market that is expected to exceed $74 billion by 20272. We now look forward to opening up clinical trial sites in the United States and additional clinical trial sites in the UK.” Neil Bell, Chief Development Officer of Avacta Life Sciences, commented: “We are excited to bring the first FAP-activated chemotherapeutic to the clinic in the US, which has the potential to meaningfully impact patients with difficult-to-treat tumours. The clearance of the AVA6000 IND by the FDA enables the opening of key US sites to support the ALS-6000-101 clinical trial which is currently recruiting patients across clinical sites in the UK.”

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Spotlight

Explore some of the key milestones in the development and engineering of monoclonal antibodies and the role they play in medicine. Learn more about drug research and development at Roche.