Shots:The US FDA has approved label expansion to Evkeeza (evinacumab-dgnb) as an adj. to diet, exercise & other lipid-lowering therapies for the treatment of children (1 to <5yrs.) with homozygous familial hypercholesterolemia (HoFH)Approval was supported by efficacy & safety results in 6 children with HoFH (incl. PK data from 4) who participated in…
Shots:Ultragenyx has completed enrolment in its P-III (Aspire) trial to assess GTX-102 (apazunersen) in Angelman syndrome pts with confirmation of full maternal UBE3A gene deletion; Aurora study in more ages & genotypes to begin in H2'25Trial will evaluate GTX-102 (intrathecal, 8mg QM for 3mos., then up to 14mg Q4M) vs a sham treatment…
Shots:Health Canada has approved label expansion of Evkeeza, as an adjunct to diet & other lipid-lowering therapies, to treat children (≥6mos.) with HoFHApproval was based on model-based analysis that predicted similar or greater LDL-C reductions with Evkeeza (15mg/kg, Q4W) in children (6mos. to 5yrs.) at 24wks. Data from compassionate use in 5 children…
Shots:The EC has approved Evkeeza (an angiopoietin-like 3 inhibitor), as an adjunct to diet & other lipid-lowering therapies, to treat children (6mos. to 5yrs.) with HoFH
Approval was based on model-based analysis that predicted similar or greater LDL-C reductions with Evkeeza (15mg/kg, Q4W) in children (6mos. to 5yrs.) at 24wks. Data from compassionate…
Shots: The EMA approved 3 BLA while 4 New Chemical Entities in December 2023, leading to treatments for patients and advances in the healthcare industryIn December 2023, the major highlighted drugs were Jempreli to treat dMMR/MSI-H Primary Advanced or Recurrent Endometrial Cancer and Zilbrysq for the treatment of Generalized Myasthenia Gravis PharmaShots has compiled a list of a…
In an interview with PharmaShots, Dr. Stanley T. Crooke, Founder, and Chief Executive Officer and Chairman of the Board at n-Lorem Foundation share his views on the collaboration with Ultragenyx.Shots:n-Lorem Foundation is the first & only foundation of its kind with a mission to provide life-saving treatments to patients who have diseases caused by extremely rare…
The US FDA has approved multiple NDAs and BLAs in Jun 2020, leading to treatments for patients and advances in the health care industry. The Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) have approved 51 novel products so far in 2020, including 8 in Jun 2020. Additionally,…
The 2020 JP Morgan Healthcare Conference was loaded with insights from key pharma companies. An analysis of events and catalysts that were announced or updated at the conference these days are included. Our PharmaShots team summarized the key deals which were presented during the updates from companies from Jan 13 to Jan 16, 2020.Date…

