As reported in PR NewsWire
; this March, Calliditas Therapeutics AB announced they have submitted a New Drug Application (NDA) for their novel oral solution, Nefecon,
to treat primary IgA Nephropathy (IgAN), to the U.S. Food and Drug Administration (FDA). They hope that the application will be granted accelerated approval, which if accepted, would make it the first approved pharmaceutical created specifically to treat IgAN.
Primary IgA Nephropathy (IgAN),
Primary IgA nephropathy (IgAN)
is a rare chronic kidney disease, progressing eventually to kidney failure. The disease, also known as Berger’s Disease, occurs after a patient experiences a buildup of antibodies in the kidneys, often as a follow-up to a viral infection in the respiratory system or gastrointestinal tracts. Males are twice as likely to develop the disease, which typically begins in adolescence, though some patients don’t show symptoms until later stages. While they make take years to show, symptoms eventually include bloody urine, loin pain, high blood pressure, lower back pain beneath the ribs, tiredness, swelling in extremities, and kidney failure. Treatments include care from a nephrologist, a kidney doctor, who may prescribe medication to lower blood pressure, to aid the kidney’s in removing fluid from the blood, to lessen inflammation, to lower cholesterol, and in severe cases, patients may undergo dialysis or kidney transplant.
The application was based on a study phase 3 study that studied the efficacy of the drug. They tested Nefecon against a placebo on 200 adult patients in a randomized, double-blind setting. The study found that their novel solution is effective at reducing proteinuria, a protein that the patient’s kidney’s fail to properly filter, and stabilizing a patients eGFR. It was well tolerated amongst patients and proved effective at meeting their primary and secondary endpoints. The company is the first to find a solution that passed through Phase 3 clinical trials to treat the IgAN.
Those working on the project are excited by the milestone and to work towards approval with the FDA. They know that if approved, this would be the first treatment for IgAN, which would address an unmet medical need. Their data is supportive, and they believe that it will aid in the approval process.
“Calliditas has long been pioneering a treatment for IgAN based on precision and disease modification that focuses on the origin of the disease, with the hope of bringing help to thousands of patients, so today is truly a special day,” said CEO Renée Aguiar-Lucander.
The company hopes the drug will receive accelerated approval, which allows their application to be expedited due to unmet needs for a serious condition. They instead approve these drugs based on a surrogate endpoint, for this trial, that was the reduction of proteinuria in the patient group in comparison to the placebo. They hope the next stage of their study will be ready in early 2021, which will provide a confirmation study to show that the drug has long-term benefits on the patients kidney functioning.
If Nefecon receives FDA approval, it will be the first IgAN therapy that was designed specifically to treat the disease, and could potentially modify the disease’s workings themselves.