Alnylam’s Onpattro Receives First-Ever US FDA Approval for Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults

US FDA approved Alnylam’s Onpattro (patisiran) lipid complex injection, a first-of-its-kind RNA interference (RNAi) therapeutic, for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. It is the first and only FDA-approved treatment for this indication. hATTR amyloidosis is a rare, inherited, rapidly progressive and life-threatening disease with a constellation of manifestations. In addition to polyneuropathy, hATTR amyloidosis can lead to other significant disabilities including decreased ambulation with the loss of the ability to walk unaided, a reduced quality of life, and a decline in cardiac functioning. In the largest controlled study of hATTR amyloidosis, Onpattro was shown to improve polyneuropathy with reversal of neuropathy impairment in a majority of patients and to improve a composite quality of life measure, reduce autonomic symptoms, and improve activities of daily living.

FDA approves Galafold, new treatment for Fabry disease,  a rare genetic disorder

The USFDA approved Galafold (migalastat), the first oral medication for the treatment of adults with Fabry disease. The drug is indicated for adults with Fabry disease who have a genetic mutation determined to be responsive to treatment with Galafold based on laboratory data. Fabry disease is a rare and serious genetic disease that results from buildup of a type of fat called globotriaosylceramide (GL-3) in blood vessels, the kidneys, the heart, the nerves and other organs. The efficacy of Galafold was demonstrated in a six-month, placebo-controlled clinical trial in 45 adults with Fabry disease. In this trial, patients treated with Galafold over six months had a greater reduction in globotriaosylceramide (GL-3) in blood vessels of the kidneys (as measured in kidney biopsy samples) as compared to patients on placebo. The safety of Galafold was studied in four clinical trials which included a total of 139 patients with Fabry disease.

FDA approves first generic drug: potassium chloride oral solution under new pathway for enhancing market competition for sole source drugs

The USFDA approved several strengths of potassium chloride oral solution as the first generic drugs to receive a Competitive Generic Therapy (CGT) designation. This new approval pathway was created to expedite the development and review of a generic drug for products that lack competition. Potassium chloride is an oral treatment that is indicated for the treatment and prevention of hypokalemia (low potassium blood levels) in patients who are on diuretics, and when dietary management with potassium-rich foods is insufficient or diuretic dose reduction is not possible.

China National Drug Administration Sets Guidelines for Overseas Drug Trial Data

China National Drug Administration (CNDA) published its Technical Guidelines for the Acceptance of Overseas Clinical Trial Data for Drugs (the Guidelines). These guidelines open the door for the registration in China of pharmaceutical drugs and medical devices that have already undergone clinical trials in other countries but previously could not be sold on the Chinese market without undergoing domestic clinical trials, allowing faster access to the Chinese market with much lower costs for pharmaceutical companies and medical device manufacturers. The guidelines are reflective of the Chinese government’s intention to increase the availability of medical devices and pharmaceutical treatments within China and to spur innovation within the domestic market for these products.

USFDA Approves First Preventative Anti-Malarial Drug, Arakoda

60 Degrees Pharmaceuticals (60P) announced the USFDA’s approval of Arakoda (tafenoquine) tablets for the prevention of malaria in patients aged 18 years and older.

For the first time in more than eighteen years, the U.S. FDA has approved a new drug for the prevention of malaria.

Tafenoquine was originally discovered by scientists at the Walter Reed Army Institute of Research (WRAIR). The approval was based on a concerted effort by the U.S. Army and 60P, involving over 21 clinical trials and over 3,100 trial subjects, to develop tafenoquine as a weekly prophylactic drug for the prevention of malaria. Arakoda provides effective protection against both of the major types of malaria (P. vivax and P. falciparum), killing the parasites in both the blood and liver.

USFDA Grants Fast Track Designation to Sesen Bio‘s Vicinium For the Treatment of Non-Muscle Invasive Bladder Cancer

Sesen Bio announced that the USFDA has granted Fast Track designation to Vicinium for the treatment of BCG-unresponsive high-grade non-muscle invasive bladder cancer (NMIBC).

Vicinium, Sesen Bio’s lead product candidate, is currently being evaluated in a Phase III registration trial, the VISTA Trial, for the treatment of patients with high-grade NMIBC who have previously received two courses of bacillus Calmette-Guérin (BCG) and whose disease is now BCG-unresponsive.

NCCN Publishes First-Ever U.S. Guidelines for Rare Cancers Associated with Pregnancy

The National Comprehensive Cancer Network (NCCN) has released new treatment guidelines for a group of rare cancers that impact women during pregnancy. Gestational trophoblastic neoplasia (GTN), also known as gestational trophoblastic disease (GTD), can occur when tumors develop in the cells that would normally form the placenta during pregnancy.

The NCCN Guidelines for GTN details treatments for several variations of the disease. For molar pregnancy (also known as a hydatiform mole, a rare mass that can form inside the womb during early pregnancy, resulting in an abnormal fetus), surgery is the first, and often only treatment required. It is generally performed via suction dilation and curettage. Low-risk GTN is primarily treated with single-agent chemotherapy, although additional chemotherapy or surgery may be required for persistent disease.

With high-risk GTN, treatment typically involves multi-agent chemotherapy, with possible radiation therapy for brain metastasis. Surgery can be used for chemotherapy-resistant disease. The NCCN Guidelines for GTN bring the total number of NCCN Guidelines to 72.

Alector’s AL001 Receives Orphan Drug Designation from USFDA for Frontotemporal Dementia

Alector announced that the US Food and Drug Administration Office of Orphan Products Development has granted orphan drug designation to AL001, a human recombinant monoclonal antibody, for the treatment of all patients with frontotemporal dementia (FTD). There is currently no approved therapy to address the underlying cause and needs of FTD patients.

FDA Initiates Steps to Encourage Opioid Use Disorder Medicine Development 

The USFDA has issued new scientific recommendations aimed at encouraging more widespread innovation and development of novel medication-assisted treatment (MAT) drugs for the treatment of opioid use disorder (OUD).

The draft guidance outlines new ways for drug developers to consider measuring and demonstrating the effectiveness and benefits of new or existing MAT products. This new draft guidance is part of the FDA’s ongoing

FDA Approves First Medicine Orkambi to Treat Underlying Cause of Cystic Fibrosis in Children

Vertex announced that the USFDA has approved Orkambi (lumacaftor/ivacaftor) to be used in children ages 2 through 5 years with cystic fibrosis (CF) who have two copies of the F508del-CFTR mutation, making it the first medicine approved to treat the underlying cause of CF in this population.

Orkambi oral granules are available in two dosage strengths (lumacaftor 100mg/ivacaftor 125mg and lumacaftor 150mg/ivacaftor 188mg) for weight-based dosing. Orkambi oral granules should be available for fulfillment within 2 to 4 weeks. This FDA approval is based on a Phase III open-label safety study in 60 patients that showed treatment with Orkambi was generally safe and well tolerated for 24 weeks, with a safety profile similar to that in patients ages 6 years and older.

Orkambi was already approved in the US for the treatment of CF in patients ages 6 and older who have two copies of the F508del-CFTR mutation. A Marketing Authorization Application (MAA) line extension for Orkambi in children ages 2 through 5 years has been submitted to the European Medicines Agency (EMA) with a decision anticipated in the first half of 2019.