Home > News > Top Stories > Rhythm Pharmaceuticals Receives FDA Approval for obesity med targeting genetic defects

Research

Rhythm Pharmaceuticals Receives FDA Approval for obesity med targeting genetic defects

Stoutness is a significant issue around the world, in any case, for a small amount of patients, hereditary changes instead of propensities are driving the sickness. With its new FDA endorsement for Imcivree, Rhythm Pharmaceuticals is venturing out treating those cases.

For a subset of heftiness patients, hereditary transformations leave them with an "voracious appetite," Rhythm CEO David Meeker, M.D., said in a meeting. Beginning stage hereditary illnesses can leave patients incapable to control corpulence with diet and exercise, and, presently, Rhythm has scored the main FDA endorsement for a focused on medication pointed toward treating them.

Imcivree, once known as setmelanotide, scored a FDA gesture to treat patients who have varieties of the POMC, PCSK1 or LEPR qualities. The medication focuses on the MC4R pathway answerable for directing yearning, and the three qualities remembered for the underlying endorsement are only an initial step, Meeker said. The organization built up a test that takes a gander at around 100 hereditary transformations, and it's trying the medication in other hereditary changes with eyes on expected endorsements down the line.

With obesity, “you can see the forest,” Meeker said, because the problem is everywhere. The challenge is finding patients who could have an underlying genetic mutations causing excessive hunger, he said, noting that “testing becomes key.”

Most people, and most doctors, still think of obesity as a “calories in, calories out,” problem, said Michael Dedekian, medical director of pediatric specialty care at Barbara Bush Children’s Hospital in Maine. Very quickly, though, the medical community’s understanding of obesity is deepening, he said. And, as that’s happening, it’s becoming clear that genetic defects can “wildly disregulate” patients’ hunger. 

With the approval and new knowledge about genetic defects, Dedekian believes the medical community is entering a “whole new era of obesity treatment." Over time, he has no doubt awareness of the genetic mutations driving some cases will increase. When abnormal hunger presents itself in young children, doctors should strongly consider a genetic test, Dedekian said.

Rhythm isn’t planning to deploy a sales force for its initial genetic mutations and will instead focus on “intensive efforts” around disease education and making its test more available, Meeker said. When doctors are confronted with a patient with abnormal hunger, they should be “thinking deep” about the cause, he added.

Spotlight

More featured news

Spotlight

Related News

PHARMA TECH

Performance Validation Joins ValGenesis Partner Program as Service Partner for the U.S. Market

PR Newswire | January 24, 2024

ValGenesis Inc., the market leader in enterprise validation lifecycle management systems (VLMS), and Performance Validation, Inc. a 100% employee-owned company serving the life sciences industry since 1988, have partnered to deliver unique compliance-focused digital validation solutions for life sciences companies in the US. Performance Validation is an industry leader with over 30 years of experience in analyzing, identifying, and testing the most critical elements of the manufacturing process for FDA-regulated industries. The company provides environmental mapping, commissioning, qualification, computer system validation, and quality system consulting services. ValGenesis is the industry innovator of digitized validation. The company's platform, which includes its flagship VLMS offering, is used in over 100,000 GMP systems worldwide, helping life sciences companies advance their digital transformation strategy, enforce compliance, and enable standardization with 100% digital, risk-based validation software. In this partnership, Performance Validation will implement and manage next-generation digital validation tools developed by ValGenesis, including ValGenesis VLMS and VLMS Express, a simplified cloud-based digital validation solution for startups and midsized companies. ValGenesis and Performance Validation are providing an integrated governance approach to further strengthen risk and compliance across their customers' manufacturing processes. "We are excited to partner with Performance Validation. Their extensive experience and our industry benchmarked digital validation platform can help life sciences companies implement their digitization plans efficiently according to Industry 4.0 standards," says Bo Olsen, ValGenesis' SVP of Partners. "By establishing a clear strategy that addresses their risk and compliance implications upfront, we can assure them of true business transformation." "Performance Validation is thrilled to embark on this collaborative journey with ValGenesis, leveraging our extensive experience and knowledge of digital validation solutions. It's a powerful combination – our hands-on validation experience and their innovative digital tools. For our clients in the life sciences, this means more efficient, streamlined, and compliant solutions. We're not just adapting to Pharma 4.0; we're helping our clients lead the way in digital transformation," says Brad Henry, Vice President of Service Lines at Performance Validation. ABOUT PERFORMANCE VALIDATION Performance Validation (PV) is a global validation partner for pharmaceutical and medical device manufacturers. Headquartered in Indianapolis, IN, we specialize in turning compressed timelines into compliant ones using innovative, adaptive approaches that balance production realities with strict regulatory requirements. Our best-in-class, cGMP-compliant services cover diverse needs from fully managed CQV to on-demand temperature mapping, smoke studies, software assurance, and more. With a dedicated team consisting of more than 95% engineers, we work closely with regulators and equipment suppliers to keep validation ahead of production curves and keep quality moving forward. ABOUT VALGENESIS ValGenesis, Inc. is the creator of an innovative software platform that serves as a foundation for managing compliance-based validation activities in life sciences companies. ValGenesis, Inc. is the provider of the first enterprise application that manages the corporate validation lifecycle process. This solution is fully compliant with U.S. FDA 21 CFR Part 11 and Annex 11 requirements. As the first fully paperless solution for electronic management of validation execution and approval, ValGenesis was selected by an industry peer review committee to receive the Parenteral Drug Association (PDA) New Innovative Technology Award in 2005.

Read More

PHARMA TECH

CGI and Körber announce global partnership to enable pharmaceutical and life sciences clients to improve production operations

PR Newswire | January 23, 2024

CGI, one of the largest business and IT consulting companies in the world, today announced a partnership with Körber aimed at enabling pharmaceutical and life sciences companies to improve their production processes by combining CGI's end-to-end services with Körber's Werum PAS-X Manufacturing Execution System (MES) Suite. The global partnership is designed to help clients increase efficiency, accuracy, and safety in pharma products and enhance decision-making through access to real-time data. "CGI works side-by-side with clients to transform data into actionable insights to drive smart, sustainable and resilient operations," said Tim Hurlebaus President, U.S. Commercial & State Government operations and global executive sponsor for CGI's health and life sciences business. "This includes combining the benefits of MES and Industry 5.0 to gather rich insights to enable operational excellence. Our partnership with Körber complements our offerings portfolio, enabling us to deliver innovative new services and solutions to support the needs of our pharma and life sciences clients." Körber's platform serves as a digital, real-time process controller to shorten time-to-market, lower production costs and enable a more patient-centric approach, improving outcomes and leading to improved health. Compliant with Good Manufacturing Practices, PAS-X MES presents a comprehensive view of data from diverse integrated systems, enabling faster and more informed decisions, which can be a critical advantage in an industry where timing and accuracy are paramount. "Our PAS-X Products and Service Suites facilitate seamless digital transformation projects at our customers globally. By combining forces with CGI's global presence, expertise and complementary service portfolio in pharma and life sciences, we can offer a unique and compelling value proposition. CGI is one of the key partners to continue to expand our offering to our global customers" says Jan-Henrik Dieckert, COO Software, Körber Business Area Pharma. Embedding robust IT and operational technology (OT) security, CGI's business consulting and MES services expertise enables faster, more seamless, and operationally integrated implementation of the platform, including transformation through technical integration, data migration, and organizational change management and adoption. "In our global client insights research, pharma and life sciences executives said optimizing operations is their top business priority. CGI serves as a trusted advisor to help them achieve this priority through ROI-led digitization investments," said Maiken Lykke, Senior Vice-President, CGI Denmark. "We work collaboratively with clients to build solutions that tailor the right mix of services and technologies for their needs. We are pleased to partner with Körber to address the unique client challenges in this industry." CGI delivers clinical and IT solutions for 11 of the top 20 global biotech and pharmaceutical companies, including helping these organizations optimize their processes; launch new therapies for cancer and rare diseases; and drive digital transformation across their research & development, manufacturing and commercial functions globally. About CGI Founded in 1976, CGI is among the largest independent IT and business consulting services firms in the world. With 91,500 consultants and professionals across the globe, CGI delivers an end-to-end portfolio of capabilities, from strategic IT and business consulting to systems integration, managed IT and business process services and intellectual property solutions. CGI works with clients through a local relationship model complemented by a global delivery network that helps clients digitally transform their organizations and accelerate results. CGI Fiscal 2023 reported revenue is C$14.30 billion. About Körber We are Körber – an international technology group with more than 12,000 employees at over 100 locations worldwide and a common goal: We turn entrepreneurial thinking into customer success and shape the technological change. In the Business Areas Digital, Pharma, Supply Chain, and Technologies, we offer products, solutions and services that inspire. We act fast to customer needs, we execute ideas seamlessly, and with our innovations we create added value for our customers. In doing so, we are increasingly building on ecosystems that solve the challenges of today and tomorrow. Körber AG is the holding company of the Körber Group.

Read More

PHARMA TECH

SHL Medical partners with SteriPack Group to set up final assembly service

PR Newswire | January 25, 2024

SHL Medical, a world-leading solutions provider of advanced drug delivery systems and SteriPack Group, renowned global supplier of secondary packing and final assembly services to the pharmaceutical industry, enter a non-exclusive strategic partnership. Together, they aim to establish a pre-validated final assembly service at the SteriPack facility in Poland, enhancing the overall flexibility of supply chain strategies for their customers. In addition to SHL Medical's existing final assembly, labeling, and packaging¬ services in the United States, this collaboration extends high-quality CMO options to Europe. Markus Puusepp, SHL Medical's Chief Growth Officer, expressed enthusiasm about the partnership, stating "This partnership is a significant addition to the Molly platform value proposition. We share common values with SteriPack, from focus on the customer to a commitment to quality, making the decision to collaborate straightforward." Recognizing the importance of flexibility and time-to-market, especially in clinical production, the partnership with SteriPack will offer SHL Medical's customers highly flexible and personalized final assembly and packaging solutions. This is particularly beneficial for small batch handling in clinical supply and niche markets like rare diseases. Emphasizing the comprehensive capabilities of the Molly platform, SHL Medical aims to deliver right-sized solutions that ensure improved service, faster timelines, and a more streamlined supply chain for its diverse customer base. John Ward, SteriPack Group's VP Pharmaceutical Solutions commented "SteriPack Group looks forward to supporting SHL Medical and their customers in providing quality and flexible solutions to a fast- moving market. With an emphasis on creating service solutions designed around specific customer requirements, this partnership will make the process of working with the Molly platform an easy decision." About SHL Medical As a world-leading solutions provider of advanced drug delivery systems, SHL Medical is the partner of choice for many leading pharma and biotech companies. Driven by its company purpose – Enabling Patients' Independence – SHL Medical offers patient-centric solutions for the design, development, and manufacturing of autoinjectors, pen injectors, as well as innovative specialty delivery systems for large-volume and high-viscosity formulations. It also offers final assembly, labeling, and packaging solutions for its drug delivery systems. In response to the rising trend in home therapy, SHL Medical has increased its developmental work in the digital healthcare sector to help improve the drug delivery ecosystem. Located across Switzerland, Taiwan, Sweden, and the US, SHL Medical's global team of experts collaborate seamlessly as one team in utilizing its comprehensive in-house manufacturing capabilities. Its solutions offer customization and optimization for each project while proactively weaving sustainability-driven measures into its designs and processes to contribute to a cleaner earth. About SteriPack Group SteriPack Group is a globally renowned contract packing and manufacturing organisation serving the pharmaceutical and medical device sectors respectively. With a strong emphasis on innovation, quality and compliance, SteriPack's services to the pharmaceutical industry include device design and development, final assembly, labelling, secondary packing, serialisation and aggregation of injectable formats including vials, pre-filled syringes, and autoinjectors. SteriPack's tailored customer-focused solutions make it an ideally suited supplier for small to medium-sized batch volumes required for a growing market of targeted therapies as well as clinical trials. Its proven capabilities in cleanroom assembly and labelling as well as secondary packing services for drug delivery systems and medical devices, combined with significant investment into growing cold chain storage capacity, have helped to make it a trusted partner to top global pharmaceutical companies.

Read More

PHARMA TECH

Performance Validation Joins ValGenesis Partner Program as Service Partner for the U.S. Market

PR Newswire | January 24, 2024

ValGenesis Inc., the market leader in enterprise validation lifecycle management systems (VLMS), and Performance Validation, Inc. a 100% employee-owned company serving the life sciences industry since 1988, have partnered to deliver unique compliance-focused digital validation solutions for life sciences companies in the US. Performance Validation is an industry leader with over 30 years of experience in analyzing, identifying, and testing the most critical elements of the manufacturing process for FDA-regulated industries. The company provides environmental mapping, commissioning, qualification, computer system validation, and quality system consulting services. ValGenesis is the industry innovator of digitized validation. The company's platform, which includes its flagship VLMS offering, is used in over 100,000 GMP systems worldwide, helping life sciences companies advance their digital transformation strategy, enforce compliance, and enable standardization with 100% digital, risk-based validation software. In this partnership, Performance Validation will implement and manage next-generation digital validation tools developed by ValGenesis, including ValGenesis VLMS and VLMS Express, a simplified cloud-based digital validation solution for startups and midsized companies. ValGenesis and Performance Validation are providing an integrated governance approach to further strengthen risk and compliance across their customers' manufacturing processes. "We are excited to partner with Performance Validation. Their extensive experience and our industry benchmarked digital validation platform can help life sciences companies implement their digitization plans efficiently according to Industry 4.0 standards," says Bo Olsen, ValGenesis' SVP of Partners. "By establishing a clear strategy that addresses their risk and compliance implications upfront, we can assure them of true business transformation." "Performance Validation is thrilled to embark on this collaborative journey with ValGenesis, leveraging our extensive experience and knowledge of digital validation solutions. It's a powerful combination – our hands-on validation experience and their innovative digital tools. For our clients in the life sciences, this means more efficient, streamlined, and compliant solutions. We're not just adapting to Pharma 4.0; we're helping our clients lead the way in digital transformation," says Brad Henry, Vice President of Service Lines at Performance Validation. ABOUT PERFORMANCE VALIDATION Performance Validation (PV) is a global validation partner for pharmaceutical and medical device manufacturers. Headquartered in Indianapolis, IN, we specialize in turning compressed timelines into compliant ones using innovative, adaptive approaches that balance production realities with strict regulatory requirements. Our best-in-class, cGMP-compliant services cover diverse needs from fully managed CQV to on-demand temperature mapping, smoke studies, software assurance, and more. With a dedicated team consisting of more than 95% engineers, we work closely with regulators and equipment suppliers to keep validation ahead of production curves and keep quality moving forward. ABOUT VALGENESIS ValGenesis, Inc. is the creator of an innovative software platform that serves as a foundation for managing compliance-based validation activities in life sciences companies. ValGenesis, Inc. is the provider of the first enterprise application that manages the corporate validation lifecycle process. This solution is fully compliant with U.S. FDA 21 CFR Part 11 and Annex 11 requirements. As the first fully paperless solution for electronic management of validation execution and approval, ValGenesis was selected by an industry peer review committee to receive the Parenteral Drug Association (PDA) New Innovative Technology Award in 2005.

Read More

PHARMA TECH

CGI and Körber announce global partnership to enable pharmaceutical and life sciences clients to improve production operations

PR Newswire | January 23, 2024

CGI, one of the largest business and IT consulting companies in the world, today announced a partnership with Körber aimed at enabling pharmaceutical and life sciences companies to improve their production processes by combining CGI's end-to-end services with Körber's Werum PAS-X Manufacturing Execution System (MES) Suite. The global partnership is designed to help clients increase efficiency, accuracy, and safety in pharma products and enhance decision-making through access to real-time data. "CGI works side-by-side with clients to transform data into actionable insights to drive smart, sustainable and resilient operations," said Tim Hurlebaus President, U.S. Commercial & State Government operations and global executive sponsor for CGI's health and life sciences business. "This includes combining the benefits of MES and Industry 5.0 to gather rich insights to enable operational excellence. Our partnership with Körber complements our offerings portfolio, enabling us to deliver innovative new services and solutions to support the needs of our pharma and life sciences clients." Körber's platform serves as a digital, real-time process controller to shorten time-to-market, lower production costs and enable a more patient-centric approach, improving outcomes and leading to improved health. Compliant with Good Manufacturing Practices, PAS-X MES presents a comprehensive view of data from diverse integrated systems, enabling faster and more informed decisions, which can be a critical advantage in an industry where timing and accuracy are paramount. "Our PAS-X Products and Service Suites facilitate seamless digital transformation projects at our customers globally. By combining forces with CGI's global presence, expertise and complementary service portfolio in pharma and life sciences, we can offer a unique and compelling value proposition. CGI is one of the key partners to continue to expand our offering to our global customers" says Jan-Henrik Dieckert, COO Software, Körber Business Area Pharma. Embedding robust IT and operational technology (OT) security, CGI's business consulting and MES services expertise enables faster, more seamless, and operationally integrated implementation of the platform, including transformation through technical integration, data migration, and organizational change management and adoption. "In our global client insights research, pharma and life sciences executives said optimizing operations is their top business priority. CGI serves as a trusted advisor to help them achieve this priority through ROI-led digitization investments," said Maiken Lykke, Senior Vice-President, CGI Denmark. "We work collaboratively with clients to build solutions that tailor the right mix of services and technologies for their needs. We are pleased to partner with Körber to address the unique client challenges in this industry." CGI delivers clinical and IT solutions for 11 of the top 20 global biotech and pharmaceutical companies, including helping these organizations optimize their processes; launch new therapies for cancer and rare diseases; and drive digital transformation across their research & development, manufacturing and commercial functions globally. About CGI Founded in 1976, CGI is among the largest independent IT and business consulting services firms in the world. With 91,500 consultants and professionals across the globe, CGI delivers an end-to-end portfolio of capabilities, from strategic IT and business consulting to systems integration, managed IT and business process services and intellectual property solutions. CGI works with clients through a local relationship model complemented by a global delivery network that helps clients digitally transform their organizations and accelerate results. CGI Fiscal 2023 reported revenue is C$14.30 billion. About Körber We are Körber – an international technology group with more than 12,000 employees at over 100 locations worldwide and a common goal: We turn entrepreneurial thinking into customer success and shape the technological change. In the Business Areas Digital, Pharma, Supply Chain, and Technologies, we offer products, solutions and services that inspire. We act fast to customer needs, we execute ideas seamlessly, and with our innovations we create added value for our customers. In doing so, we are increasingly building on ecosystems that solve the challenges of today and tomorrow. Körber AG is the holding company of the Körber Group.

Read More

PHARMA TECH

SHL Medical partners with SteriPack Group to set up final assembly service

PR Newswire | January 25, 2024

SHL Medical, a world-leading solutions provider of advanced drug delivery systems and SteriPack Group, renowned global supplier of secondary packing and final assembly services to the pharmaceutical industry, enter a non-exclusive strategic partnership. Together, they aim to establish a pre-validated final assembly service at the SteriPack facility in Poland, enhancing the overall flexibility of supply chain strategies for their customers. In addition to SHL Medical's existing final assembly, labeling, and packaging¬ services in the United States, this collaboration extends high-quality CMO options to Europe. Markus Puusepp, SHL Medical's Chief Growth Officer, expressed enthusiasm about the partnership, stating "This partnership is a significant addition to the Molly platform value proposition. We share common values with SteriPack, from focus on the customer to a commitment to quality, making the decision to collaborate straightforward." Recognizing the importance of flexibility and time-to-market, especially in clinical production, the partnership with SteriPack will offer SHL Medical's customers highly flexible and personalized final assembly and packaging solutions. This is particularly beneficial for small batch handling in clinical supply and niche markets like rare diseases. Emphasizing the comprehensive capabilities of the Molly platform, SHL Medical aims to deliver right-sized solutions that ensure improved service, faster timelines, and a more streamlined supply chain for its diverse customer base. John Ward, SteriPack Group's VP Pharmaceutical Solutions commented "SteriPack Group looks forward to supporting SHL Medical and their customers in providing quality and flexible solutions to a fast- moving market. With an emphasis on creating service solutions designed around specific customer requirements, this partnership will make the process of working with the Molly platform an easy decision." About SHL Medical As a world-leading solutions provider of advanced drug delivery systems, SHL Medical is the partner of choice for many leading pharma and biotech companies. Driven by its company purpose – Enabling Patients' Independence – SHL Medical offers patient-centric solutions for the design, development, and manufacturing of autoinjectors, pen injectors, as well as innovative specialty delivery systems for large-volume and high-viscosity formulations. It also offers final assembly, labeling, and packaging solutions for its drug delivery systems. In response to the rising trend in home therapy, SHL Medical has increased its developmental work in the digital healthcare sector to help improve the drug delivery ecosystem. Located across Switzerland, Taiwan, Sweden, and the US, SHL Medical's global team of experts collaborate seamlessly as one team in utilizing its comprehensive in-house manufacturing capabilities. Its solutions offer customization and optimization for each project while proactively weaving sustainability-driven measures into its designs and processes to contribute to a cleaner earth. About SteriPack Group SteriPack Group is a globally renowned contract packing and manufacturing organisation serving the pharmaceutical and medical device sectors respectively. With a strong emphasis on innovation, quality and compliance, SteriPack's services to the pharmaceutical industry include device design and development, final assembly, labelling, secondary packing, serialisation and aggregation of injectable formats including vials, pre-filled syringes, and autoinjectors. SteriPack's tailored customer-focused solutions make it an ideally suited supplier for small to medium-sized batch volumes required for a growing market of targeted therapies as well as clinical trials. Its proven capabilities in cleanroom assembly and labelling as well as secondary packing services for drug delivery systems and medical devices, combined with significant investment into growing cold chain storage capacity, have helped to make it a trusted partner to top global pharmaceutical companies.

Read More

More featured news