Search Results for: ORPHAN DRUG MARKET Drug Developers
Articles
ORPHAN DRUG MARKET - Drug Developers Drawn to Orphan Drugs Market: Financial Incentives Create Rich, Competitive Pipelines November 17, 2014
As drug developers abandon the “blockbuster model” in favor of greater focus on drug development for rare conditions, the global orphan drugs market is becoming increasingly competitive. It is imperative that drug developers continually keep a tab of competitors’ pipelines as approval and reimbursement of new orphan drugs are highly dependent on the availability of alternative therapies.
Terns Pharmaceuticals Announces Orphan Drug Designation Granted to TERN-701 for the Treatment of Chronic Myeloid Leukemia March 13, 2024
Terns Pharmaceuticals, Inc. recently announced the US FDA granted Orphan Drug Designation for TERN-701 for the treatment of chronic myeloid...NovelMed's Ruxoprubart Receives FDA Orphan Drug Designation for Paroxysmal Nocturnal Hemoglobinuria Treatment February 12, 2024
NovelMed recently announced the US FDA has granted Orphan Drug Designation (ODD) to Ruxoprubart, an alternative pathway (AP) blocker anti-Bb...DRUG DEVELOPMENT - Overcoming Challenges on the Path From Candidate Selection to First-in-Human Clinical Testing June 5, 2023
Eleanor Row, PhD, explores the current challenges drug developers face in transitioning their candidate molecules to first-in-human (FIH) clinical trials, including what the different drug development functions should be considering at this stage, and how an integrated approach can help to accelerate your molecule’s pathway to trial and beyond.
MetrioPharm Receives Orphan Drug Designation for the Treatment of Duchenne Muscular Dystrophy May 30, 2023
MetrioPharm AG recently announced the US FDA has granted Orphan Drug Designation for MP1032 for the treatment of Duchenne muscular...ROUNDTABLE DISCUSSION - Which Trends Will Have the Most Impact on Drug Development in 2023? January 16, 2023
Contributor Cindy H. Dubin posed this question to life science leaders during a recent roundtable discussion, and one common theme is the focus on sustainability in pharmaceutical development.
DRUG DELIVERY - Using the IPEG™ Delivery System to Topically Treat Rare Diseases in Dermatology While Limiting Systemic Absorption November 30, 2022
Zachary Rome, Jessica Raiz, MPH, and Alan M. Mendelsohn, MD, are advancing innovative clinical research that is evaluating high-potential, low-risk treatments for rare dermatologic diseases using unique topical vehicles combined with established medications that enable the targeted delivery of therapies to the epidermis and dermis while minimizing systemic absorption.
Pace Life Sciences Acquires Biopharma Global, Expanding FDA Regulatory Affairs Strategy & Consulting Capabilities to the Biotechnology & Pharmaceutical Markets September 8, 2022
Pace Life Sciences, LLC recently announced it has acquired Biopharma Global (Biopharma). Biopharma specializes in full-service regulatory support for orphan...SPECIAL ROUNDTABLE - Which Trends Will Have the Most Impact on Drug Development in 2022? June 6, 2022
Drug Development & Delivery posed this question to life science leaders during a recent roundtable discussion. While their responses are varied, a common theme is that COVID-19 has left a lasting impact on the industry….
SPECIAL FEATURE - PFS & Parenteral Manufacturing: How COVID-19 Changed the Market May 3, 2021
Contributor Cindy Dubin explores how device developers and parenteral contract development and manufacturing organizations (CDMOs) are addressing current challenges, as well as advancements in customized device design, the continuous effort to incorporate safety and human factors, and how COVID-19 is shaping the future of the market.
DRUG DEVELOPMENT - Understanding & Targeting the Mechanism of Action of Developmental Disorders March 1, 2021
Michael Snape, PhD, says when identifying promising therapeutic targets for any disease or condition, including developmental disorders such as Fragile X syndrome, PMS, CDM1, and Rett syndrome, it is critical researchers work to understand the mechanism of disease and disease pathways as well as the unmet need and patient experience.
MULTIPARTICULATE FORMULATIONS - Using Multiparticulate Technology to Develop Pediatric Drug Products February 28, 2019
Sven Stegemann, PhD, Matt Shaffer, Samantha Saville, and Jaspreet Arora, PhD, believe as pediatric formulations are expected to continue as a core research area in pharmaceutical technology, MP technologies have the potential to play a key role.
TRANSLATIONAL PHARMACEUTICS - Accelerating Early-Stage Drug Development With Integrated CDMO & CRO Services January 9, 2019
Peter Scholes, PhD, describes the current challenges and questions faced by drug developers in accelerating molecules to POC and developing optimized and scalable drug products for patient trials.
SPECIAL FEATURE - Injection Devices: As Self-Injection Grows, Drug Delivery Gets Smarter August 28, 2018
Contributor Cindy H. Dubin reviews some of the innovative and novel technologies developers are deploying to make self-injection easier and improve patient adherence.
CONTRACT MANUFACTURING - Flexibility in an Evolving Market April 3, 2017
Matthew Moorcroft, PhD, warns that in the fast-moving pharmaceutical industry, change is the only constant. And to keep up with market developments, small molecule manufacturers must first understand the trends.
SPECIAL FEATURE - Injectable Drug Delivery: Key Trends Define Device Design Now & in the Future August 31, 2016
Contributor Cindy H. Dubin spoke with some of the world’s leading device developers about their current injection technologies and how their devices are addressing the current trends and opportunities in the industry.
Orphan Drug Prices Facing Increased Scrutiny Following Historic Year for Approvals January 20, 2015
With the US Food and Drug Administration (FDA) approving an all-time record number of orphan drugs during 2014, the pricing...Latest Frost Reports: Next-Generation Therapeutics for Infectious Diseases; Orphan Drugs Market March 31, 2014
Next-Generation Therapeutics The available antivirals for commonly occurring infections, such as respiratory syncytial virus (RSV) and influenza are characterized by...PHARMACEUTICAL GLOBALIZATION - Navigating Opportunities & Obstacles December 4, 2023
Jürgen Hönig says rapid globalization of the pharmaceutical industry has created new complexities as companies navigate different regulations and government policies, but there are many reasons for optimism, including harmonization, collaboration, and convergence.
EPO Decision to Grant European Patent for Rencofilstat Further Strengthens Hepion’s Patent Portfolio March 2, 2023
Hepion Pharmaceuticals, Inc. recently announced the European Patent Office (EPO) has granted European Patent No. EP 3886813, covering the innovative...
