FDA lifts clinical hold on Fitusiran

Alnylam announces lift of FDA clinical hold on Fitusiran

Alnylam announces lift of FDA clinical hold on Fitusiran. Stockwinners.com
FDA lifts clinical hold on Fitusiran

Alnylam Pharmaceuticals (ALNY) and Sanofi Genzyme, the specialty care global business unit of Sanofi (SNY), announced today that the U.S. Food and Drug Administration has lifted the hold on clinical studies with fitusiran, including the Phase 2 open-label extension study and the ATLAS Phase 3 program.

Alnylam and the FDA had previously reached alignment on new clinical risk mitigation measures, including protocol-specified guidelines and additional investigator and patient education concerning reduced doses of replacement factor or bypassing agent to treat any breakthrough bleeds in fitusiran studies.

The FDA has now approved the protocol amendments and other updated clinical materials for fitusiran studies.

Fitusiran is an investigational RNAi therapeutic targeting antithrombin for the treatment of patients with hemophilia A and B.

It is designed to lower levels of AT with the goal of promoting sufficient thrombin generation to restore hemostasis and prevent bleeding.

Alnylam suspended patient dosing in all ongoing studies of its RNA therapy fitusiran to treat hemophilia A or B, after a patient died of swelling in the brain in a Phase 2 open-label extension trial.

Fitusiran, also known as ALN-AT3SC, is designed to specifically lower levels of antithrombin — a protein that inhibits clotting — to improve production of thrombin, a clotting factor. As such, it aims to create a better balance of clotting components that would prevent bleeding events in hemophilia A and B patients.

Alnylam and the FDA had previously reached alignment on new clinical risk mitigation measures, including protocol-specified guidelines and additional investigator and patient education concerning reduced doses of replacement factor or bypassing agent to treat any breakthrough bleeds in #fitusiran studies.

The FDA has now approved the protocol amendments and other updated clinical materials for fitusiran studies.

ALNY closed at $123.66. It last traded at $128.10. SNY closed at $43.09.


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Aclaris Therapeutics receives FDA approval for Eskata

Aclaris Therapeutics receives FDA approval for Eskata topical solution

FDA approval for Eskata topical solution. Stockwinners
   FDA approves Eskata topical solution

Aclaris Therapeutics (ACRS) announced that the FDA has approved Eskata topical solution, 40% for the treatment of raised seborrheic keratoses, or SKs. SKs are non-cancerous skin growths that affect more than 83 million American adults and can be an aesthetic skin concern.

Seborrheic keratosis  is a common skin growth. It may seem worrisome because it can look like a wart, pre-cancerous skin growth (actinic keratosis), or skin cancer. Despite their appearance, seborrheic keratoses are harmless.

Most people get these growths when they are middle aged or older. Because they begin at a later age and can have a wart-like appearance, seborrheic keratoses are often called the “barnacles of aging.” It’s possible to have just one of these growths, but most people develop several. Some growths may have a warty surface while others look like dabs of warm, brown candle wax on the skin.

SKs tend to increase in size and number with age. The condition is more prevalent than acne, psoriasis and rosacea combined.

The FDA approval of Eskata is based on two pivotal Phase 3 trials that demonstrated the safety and efficacy of Eskata for the treatment of raised SKs.

In these trials, patients received up to two treatments with #Eskata, with one at treatment initiation and a second at week three.

Patients treated with Eskata were more likely to have all four treated SKs completely cleared after two treatments than patients who received placebo.

Treatment with Eskata was generally well tolerated, with the most common side effects being itching, stinging, crusting, swelling, redness and scaling at the site of application.

ACRS closed at $24.72.


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