Relyvrio, a New Treatment Option for ALS, Gets FDA Approval
Relyvrio is an oral, fixed-dose coformulation of sodium phenylbutyrate and taurursodiol.
Amyotrophic Lateral Sclerosis
MPR Weekly Dose Podcast #130
This week, news of the FDA’s vote on AMX0035, the novel ALS treatment; CPAP and BiPAP device recalls; Orphan Drug designation for a novel CRISPR therapy; Imfinzi gains new approval; and Spevigo is approved for pustular psoriasis flares.
FDA Panel Votes in Favor of Fixed-Dose Combination Therapy for ALS
AMX0035 is an oral, fixed-dose coformulation of sodium phenylbutyrate and taurursodiol, also known as ursodoxicoltaurine.
Tofersen Gets Priority Review for Superoxide Dismutase 1 Amyotrophic Lateral Sclerosis
Tofersen is an investigational antisense medicine that reduces the synthesis of SOD1 protein production by binding to SOD1 mRNA, allowing for its degradation by RNase-H.
June 2022 Recap: Drug Pipeline Updates
Stay up-to-date on the latest drugs in development.
Oral Treatment Radicava ORS Now Available for Patients With ALS
The oral suspension can be administered by mouth or via feeding tube using a 5mL oral syringe that comes with the product.
FDA Extends Review Period for Fixed-Dose Combo Therapy for ALS
The FDA extended the action date by 3 months to allow more time to review additional analyses of data from the Company’s clinical studies.
Oral Suspension Formulation of Edaravone Approved for ALS
The approval of Radicava ORS was based on a bioavailability study and a safety study.
FDA Panel Votes on Fixed-Dose Combination Therapy for ALS
AMX0035 is an oral, fixed-dose coformulation of sodium phenylbutyrate and taurursodiol, also known as ursodoxicoltaurine.
Oral Formulation of Edaravone Gets Priority Review for ALS
Oral edaravone was developed to have a similar clinical profile as the intravenous formulation Radicava.
