Aligos Therapeutics’ Post

“About 60-70 percent of the products we regulate are for rare diseases. We're up to 10,000 rare diseases impacting millions of individuals in the U.S.” - Dr. Peter Marks, Center for Biologics Evaluation and Research, FDA Dr. Marks words during the Reagan-Udall Foundation for the FDA workshop echo those of the 300+ million people worldwide affected by a #RareDisease. Learn more about the impact of rare diseases at https://lnkd.in/gUuRcEWs.

Breakthrough in Hepatitis Treatment: FDA Grants Breakthrough Therapy Designation to DURECT Corporation's Larsucosterol DURECT Corporation announced that the U.S. FDA has granted Breakthrough Therapy Designation to larsucosterol, a promising treatment for severe alcohol-associated hepatitis (AH), based on positive Phase IIb AHFIRM trial results. The trial demonstrated safety and efficacy, with a significant reduction in 90-day mortality for patients. This designation highlights the potential of larsucosterol to address the high mortality rate of AH, which currently lacks approved treatments. DURECT Corporation plans to advance larsucosterol to a Phase III trial, aiming to establish its effectiveness further. The drug's development and potential approval could provide a life-saving option for AH patients, significantly improving patient outcomes. For more details please click the link! https://lnkd.in/dCZhbVEz #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical 

In a comparison of thrombotic adverse events in patients treated with factor VIII products and emicizumab, thrombotic AEs in the United States Food and Drug Administration Adverse Event Reporting System data were about 3 times more frequently reported for emicizumab than for FVIII products. More research and efforts in the future are warranted for monitoring, elucidating, and preventing the potential risk of thrombotic AEs in hemophilia therapy, including emicizumab. https://lnkd.in/gdcyKPFc Hyunjeong Cho | Ki Young Yoo | Ju-Young Shin | Eun-Kyoung Lee | BongKyoo Choi

#Bronchiectasis is a chronic condition affecting approx. 370,000 adults in the US, where the lung’s bronchi become permanently damaged. On #WorldBronchiectasisDay, we are raising awareness for non-cystic fibrosis bronchiectasis (NCFB), a disease with no FDA-approved drugs specifically for NCFB. Verona’s investigational drug #ensifentrine is currently in Phase 2 clinical studies for the treatment of NCFB. Learn more about this focus area: https://bit.ly/3z3gVvX

Unraveling the Mystery: Immunological Mechanisms Behind Cutaneous Adverse Drug Reactions Discover the intricate immunological processes driving cutaneous adverse drug reactions. Explore the mysteries unravelled in this insightful study. Learn More: https://lnkd.in/gm_7g585

STAT+: Pharmalittle: We’re reading about Zepbound trial results, an Alnylam heart drug, and more

STAT+: Pharmalittle: We’re reading about Zepbound trial results, an Alnylam heart drug, and more

Part I: Rare Disease Drug Development: What patients and Advocates need to know. by National Organization for Rare Disorders FDA Foundation Fighting Blindness #raredisease #LCA10 #orphandrugs https://lnkd.in/dty4c4_Y

Interesting round up here of the movement, new developments and the future outlook in the orphan drug space.

US Government Accountability Office 63K followers - 9/29/23 This report covers incentives and policy options to help develop antiviral drugs now, should policymakers want to invest in these drugs to prepare for a future pandemic. #publichealth #antiviral #drugdiscovery #drugs #publichealthresearch #drugdevelopment