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Akebia Recovers From Rejection to Win FDA Approval for Anemia Drug

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An Akebia Therapeutics drug for a type of anemia has won FDA approval, a regulatory decision that comes nearly two years after the agency rejected the drug due to safety concerns.

Safety remains a concern about the small molecule, vadadustat. The drug’s label carries a black box warning about an increased risk of death from a variety of cardiovascular complications. But the Akebia product, which will be branded as Vafseo, now introduces a new treatment option for the estimated 500,000 adults in the U.S who suffer from anemia due to chronic kidney disease (CKD). To use this drug, patients must have been receiving dialysis for at least three months.

Anemia develops when there aren’t enough healthy red blood cells to carry oxygen to tissues in the body. It’s common in chronic kidney disease patients because their kidneys do not produce enough erythropoietin, a hormone that helps regulate red blood cell production. This anemia can be treated with erythropoietin-stimulating agents, engineered versions of the hormone administered as chronic injections.

Akebia’s Vafseo offers more convenient oral dosing. This drug belongs to a class of drugs called hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitors. By mimicking the effects of high altitude on the body, these drugs get the body to produce erythropoietin. When the FDA rejected this drug in 2022, it flagged cardiovascular risks and asked Akebia to run another clinical trial. Instead, the company resubmitted an application with additional post-marketing safety data from Japan, where the drug has been used since its approval there in 2020.

Vafseo will compete against GSK’s Jesduvroq, which last year became the first FDA-approved oral medication for anemia caused by CKD. Jesduvroq is limited to patients who have been on dialysis for at least four months. Similar to Akebia’s drug, Jesduvroq’s label carries a black box warning for cardiovascular risks. Akebia said it will commercialize Vafseo in the U.S. with its established commercial team, which has renal experience and a relationship with CSL Vifor, the kidney disease-focused company that is the product of CSL Limited’s 2021 acquisition of Vifor Pharma.

Here’s a recap of other recent regulatory news:

—Winrevair, the pulmonary arterial hypertension (PAH) drug that was the centerpiece of an $11.3 billion Merck acquisition, won FDA approval. The PAH drugs already available treat symptoms. Winrevair is the first drug that addresses an underlying cause of this disorder affecting heart and lung function.

—In other PAH news, the FDA approved Johnson & Johnson’s Opsynvi. This drug combines two older PAH drugs, macitentan and taladifil. Macitentan blocks the endothelin receptor while taladifil is a phosphodiesterase 5 inhibitor. Addressing both pathways requires patients to take multiple pills. Opsynvi is the first drug that combines both mechanisms in a single once-daily pill.

—AstraZeneca’s drug Ultomiris added neuromyelitis optica spectrum disorder (NMOSD) as a new indication for the blockbuster drug. NMOSD is an autoimmune disease affecting the central nervous system, including the spine and optic nerves. Ultomiris blocks C5, a protein of the complement system, a part of the immune system. The drug has approvals for treating several other rare, complement system disorders.

—Metabolic dysfunction-associated steatohepatitis (MASH), a fatty liver disease that affects as many as 7 million Americans, now has its first approved drug. The FDA gave the green light to Rezdiffra, a Madrigal Pharmaceuticals’ medication that posted clinical trial results showing it can reverse the liver scarring that is characteristic of the disorder. Madrigal’s once-daily pill carries an annual wholesale price of $47,400.

—An Italfarmaco drug for Duchenne muscular dystrophy won FDA approval for treating all patients who have the inherited muscle-wasting disorder, regardless of the genetic variant driving their disease. Corticosteroids are a standard first-line therapy for Duchenne. The Italfarmaco drug, Duvyzat, is the first nonsteroidal therapy approved for the disease.

—Orchard Therapeutics gene therapy Lenmeldy is now the first FDA-approved treatment for metachromatic leukodystrophy (MLD), a rare enzyme deficiency. Lenmeldy is made by modifying a patient’s hematopoietic stem cells to carry a functional gene that codes for the deficient enzyme. Orchard is now part of Japanese drugmaker Kyowa Kirin following a $387 million acquisition announced last year.

—Regeneron Pharmaceuticals’ drug odronextamab was turned down by the FDA for two indications: follicular lymphoma and diffuse large B-cell lymphoma. According to Regeneron, the agency did not flag any safety, efficacy, or manufacturing issues for the bispecific antibody. The FDA told Regeneron it cannot resubmit applications until confirmatory studies are underway and completion timelines are agreed upon. Regeneron said it will share updates on enrollment and regulatory timelines later this year. The drug is still under regulatory review in Europe.

—The FDA awarded full approval to AbbVie ovarian cancer drug Elahere. The antibody drug conjugate developed by ImmunoGen won accelerated approval in 2022. AbbVie acquired ImmunoGen last year in a $10.1 billion deal.

—Invivyd antibody drug Pemgarda was granted emergency use authorization for prevention of Covid-19 in adults and adolescents with moderate-to-severe compromised immune systems. Other Covid-19 antibody drugs have had their authorizations revoked as they proved ineffective against new variants. Pemgarda comes from an Invivyd platform technology designed to address rapid viral evolution.

—Idorsia Pharmaceuticals’ aprocitentan, brand name Tryviao, received FDA approval for treating hypertension when used in combination with other antihypertension drugs. The once-daily pill is designed to block the endothelin receptor. It’s the first approved high blood pressure drug with this mechanism of action.

—Takeda Pharmaceutical drug Iclusig is now FDA approved for treating Philadelphia chromosome-positive acute lymphoblastic leukemia when used in combination with chemotherapy. The once-daily pill works by blocking enzymes associated with cancer growth. The accelerated approval adds to the list of indications for the drug, which is used to treat three types of leukemia.

—The European Commission granted marketing authorization to Prevnar 20 for preventing pneumococcal disease in infants and children. The conjugate vaccine, which is designed to protect against the 20 circulating strains responsible for most pneumococcal infections, won European approval for adults in 2022. Prevnar 20 was first approved by the FDA for adults in 2021 and for children last year.

—BeiGene cancer immunotherapy Tevimbra received a long-awaited FDA approval for treating adults with advanced esophageal squamous cell carcinoma. The FDA decision expected in 2022 was delayed by Covid-19. Though the drug received European Commission approval last year, BeiGene lost Novartis as a partner on the cancer therapy along the way. BeiGene said it expects Tevimbra, part of the class of drugs known as checkpoint inhibitors, will become available in the U.S. in the second half of this year.

—Mirum Pharmaceuticals drug Livmarli landed FDA approval for treating pruritus, or severe itching, caused by the rare liver disease progressive familial intrahepatic cholestasis (PFIC). The approval cover patients age 5 and older. The company has also submitted an application seeking approval of a higher concentration formulation for treating younger PFIC patients. Livmarli was first approved by the FDA in 2021 as a treatment for pruritus caused by Alagille syndrome.

Public domain image by Flickr user SciTechTrend

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