FDA
-
Ionis Rare Disease Drug Is Headed to FDA, But Forthcoming Data Will Decide Competitive Profile
In its Phase 3 test, Ionis Pharmaceuticals drug donidalorsen reduced the frequency of swelling attacks caused by the rare disease hereditary angioedema. If Ionis can commercialize this drug, competition would include products from Takeda Pharmaceutical and BioCryst Pharmaceuticals.
-
Healthcare Docket: Will Florida’s Plan to Import Drugs From Canada Open the Floodgates?
No matter the outcome, it’s certain that the cost of drugs will not be an issue easily resolved anytime soon even though several other states also have laws allowing for drug imports.
-
Do you advise self-insured employers? You Can Help Us!
Take part in this survey and share some of the trends you are seeing among your clients across healthcare, including chronic conditions, behavioral health and navigation.
-
Amgen Osteoporosis Drug Prolia Is Hit With FDA’s Strictest Warning
The FDA said Amgen’s Prolia may lead to low levels of calcium, or hypocalcemia. The osteoporosis drug’s black box warning follows an FDA review of Centers for Medicare & Medicaid Services studies that found Prolia led to a significant increase in the risk of severe hypocalcemia compared to treatment with an older class of medicines.
-
Vertex’s CRISPR Gene Therapy Lands Another FDA Nod in a Rare Blood Disease
The FDA approved Vertex Pharmaceuticals’ gene therapy Casgevy for treating beta thalassemia, an inherited blood disease that leads to low levels of functioning hemoglobin. Last month, the one-time treatment won its first FDA nod for treating sickle cell disease.
-
Manufacturing Issues Block Astellas From a First-in-Class Cancer Drug Approval
Astellas Pharma’s zolbetuximab was on track to become the first gastric cancer drug that targets claudin 18.2, a protein found on cancerous stomach cells. Others with clinical-stage programs addressing this target include AstraZeneca and Bristol Myers Squibb.
-
FDA’s Preliminary Review of GLP-1 Meds Finds No Tie to Suicidal Thoughts, Actions
The FDA has found no clear relationship between GLP-1 drugs, such as Ozempic and Mounjaro, and reports of suicidal thoughts or actions. But the agency added that it can’t definitively rule out the risk, and its review is ongoing.
-
FDA Issues Alert to Mitigate Risks to a Widely Used Allergy, Nausea Drug
The FDA said promethazine hydrochloride, a generic drug used to manage allergies and motion sickness, should be administered by intramuscular injection. Intravenous injection can cause chemical irritation and tissue damage, an FDA alert states.
-
Chiesi Rare Skin Disease Drug Acquired in Billion Dollar Deal Wins FDA Approval
Chiesi Group’s Filsuvez is the second drug to win FDA approval for epidermolysis bullosa and the first for junctional EB, a more severe form of the rare skin disease. The birch tree bark-derived drug is from Chiesi’s $1.25 billion Amryt Pharma acquisition.
-
FDA Approves Not One, But Two Sickle Cell Disease Gene Therapies
The first gene therapies for sickle cell disease are now approved: Casgevy from Vertex Pharmaceuticals Casgevy and Lyfgenia from Bluebird Bio. Despite the same-day approval, key differences give one of these therapies commercialization advantages over the other.
-
Trayt Health Seeks to Increase Access to Diagnoses and Treatments
CEO Malekeh Amini explains how Trayt Health can bridge the gap for patients seeking neurological care.
-
Novartis Nabs FDA Nod for Rare Disease Drug Rival to AstraZeneca Meds
FDA approval of Novartis’s Fabhalta makes the drug the first approved oral therapy for rare blood disorder paroxysmal nocturnal hemoglobinuria. With clinical data showing superiority versus two infused AstraZeneca drugs, Novartis’s pill is well-positioned to take market share from those blockbuster products.
-
Devices & Diagnostics, Health Tech, Startups
Ketryx Rakes In $14M to Speed Up Medical Device Development & Quality Assurance
Ketryx raised $14 million in Series A funds. The startups sells a platform — designed around FDA regulations and quality standards — that automates much of the work required from medical device companies’ development and quality teams.
-
Elli Lilly Cancer Drug Lands Its Second FDA Approval This Year
Eli Lilly’s Jaypirca is now approved for treating three types of blood cancers. While Jaypirca blocks a target already addressed by other cancer therapies, the Lilly molecule does it in a way that sets it apart from the rest of its drug class.
-
FDA Probes New Cases of Cancer That May Stem From Cancer Cell Therapies
CAR T-therapies for cancer are now under tighter FDA scrutiny as the agency opens an inquiry regarding reports that some patients who received these cell therapies went on to develop new cases of cancer. The FDA said benefits of these therapies still outweigh their risks, but it is evaluating the need for regulatory action.
-
FDA Approval of SpringWorks Drug Is the First for Rare Connective Tissue Tumor
SpringWorks Therapeutics drug Ogsiveo is now FDA-approved for treating desmoid tumors, a type of tumor affecting connective tissue. The regulatory decision makes the pill the first therapy approved specifically for treating this rare disorder.
-
AstraZeneca Cancer Drug Is First in Class, But FDA Nod Is Narrower Than Expected
The FDA approved AstraZeneca’s Truqap as a new treatment for advanced cases of breast cancer. The drug is the first in a new class of therapies addressing AKT genetic alterations.