Alzheimer’s Therapy Is Coming to the Home With FDA Approval of Injectable Eisai, Biogen Drug

The Eisai- and Biogen-partnered Alzheimer’s disease drug Leqembi has been available to date as an intravenous infusion that takes about an hour. Patients will soon have the option to choose an injectable version that can be dosed in 15 seconds.

The new FDA-approved injectable formulation is only for maintenance dosing; patients still must start with intravenously infused Leqembi every two weeks along with the standard monitoring that comes with it. But after 18 months, patients may switch to a new weekly injectable formulation of Leqembi for maintenance therapy, according to the approval announcement released late Friday just ahead of the Labor Day holiday weekend.

The new Eisai and Biogen injectable drug will carry the brand name Leqembi Iqlik (pronounced “I click”). Administered via an auto-injection pen, dosing may be done at home, easing the burden on both patients and their caregivers. Eisai and Biogen are aiming for an Oct. 6 launch of Iqlik.

While IV dosing of Leqembi takes about an hour, set-up time and the required post-infusion observation period means patients and their caregivers must plan to be at an infusion center for several hours. Patients must be monitored for adverse effects of the drug, an antibody that clears amyloid plaques. Bleeding and swelling is a known and potentially fatal adverse effect of this class of Alzheimer’s drugs. Clinicians monitor for this complication, called amyloid-related imaging abnormalities with edema (ARIA-E), through MRI scans starting with the fifth infusion. Last Thursday, the FDA issued a safety communication recommending MRI scanning before the third infusion to potentially identify ARIA-E even earlier. This change is now reflected in the drug’s updated label.

Iqlik’s FDA submission was based on the results of a Phase 3 open-label extension study that evaluated a range of subcutaneous doses in patients with early Alzheimer’s. Data showed that transitioning from IV-infused Leqembi to the injectable version of the drug at 18 months maintained clinical and biomarker benefits that were comparable to continued IV dosing. While the safety of the injectable formulation was similar to that of IV maintenance treatment, Eisai highlighted one key difference: systemic reactions were much less common with injectable dosing.

Leqembi and Leqembi Iqlik stem from a multi-drug Alzheimer’s alliance between Eisai and Biogen. The two companies share in development and commercialization of the partnered drugs. While Biogen led development of the now defunct Alzheimer’s drug Aduhelm, Eisai led development of Leqembi, which secured full FDA approval in 2023.

Iqlik carries a list price of $375 per autoinjector, which works out to $19,500 a year. By comparison, Eisai initially set a $26,500 list price for infused Leqembi. In a statement issued about Iqlik’s pricing, Eisai noted that under Medicare Part D currently patients have a $2,000 cap on their total annual out-of-pocket spending on covered drugs. That means beneficiaries will pay no more than $2,000 a year, and perhaps even less, Eisai said.

Dr. Howard Fillit, co-founder and chief science officer of the Alzheimer’s Drug Discovery Foundation, views subcutaneous Leqembi as a crucial to making the drug more accessible to patients, moving the field of Alzheimer’s therapy toward the bypassing of infusions altogether.

“This milestone lessens the burden on patients and caregivers by reducing the logistical challenges of receiving Alzheimer’s treatment, while also bringing us closer to the day when patients can more easily receive a combination of drugs, potentially administered from home,” Fillit said in an emailed statement.

The FDA issued several other notable regulatory approvals to close out August. Here’s a recap of those decisions and other recent regulatory moves:

Regulatory Approvals…

—Sanofi’s immunology portfolio is adding a projected blockbuster seller with the FDA approval of rilzabrutinib, brand name Wayrilz, for the treatment of adults with chronic immune thrombocytopenia (ITP), a rare immune disorder leading to low platelet levels. Wayrilz is an oral small molecule inhibitor of an enzyme called Bruton’s tyrosine kinase (BTK). The FDA decision for the twice-daily pill makes it the first BTK inhibitor approved in the U.S. for ITP. The drug came to Sanofi as part of the pharma giant’s $3.7 billion acquisition of Principia Biopharma in 2020.

—The FDA approved updated messenger RNA Covid-19 vaccines from partners Pfizer and BioNTech as well as Moderna, but for narrower use than last year’s shots. The approval of both vaccines covers adults age 65 and older. For younger people — age 5 to 64 for Pfizer and BioNTech’s Comirnaty and age 12 to 64 for Moderna’s Spikevax — the approvals cover those who have at least one underlying condition placing them at increased risk of severe outcomes from a Covid-19 infection. Previously, these vaccines were approved for broader populations. The latest approvals for Comirnaty and Spikevax are in line with the FDA’s May approval of Novavax’s protein-based Covid vaccine, Nuvaxovid, which is being marketed by Sanofi under a deal struck last year.

—Startup ExCellThera received conditional marketing authorization in Europe for Zemcelpro, a cord blood-derived cell therapy for blood cancers. The approval specifically covers patients unable to find a matched donor for a stem cell transplant, a standard treatment for such cancers.

—The European Commission approved Gilead Sciences lenacapavir, a twice-a-year-injection, as a pre-exposure prophylaxis drug for HIV. It will be marketed in Europe under the brand name Yeytuo. In the U.S., the shot is marketed under the brand name Yeztugo following its FDA approval in June. The drug is still under regulatory review in several more countries.

—FDA approval of Ionis Pharmaceuticals drug Dawnzera gives the biotech entry into the crowded market for the rare disease hereditary angioedema. But Ionis aims to stand out with clinical data showing patients who switched from other drugs achieved even better reduction in the swelling attacks caused by this rare disease.

—The European Commission approved Madrigal Pharmaceutical’s Rezdiffra for the treatment of metabolic dysfunction-associated steatohepatitis, the fatty liver disease called MASH. The conditional marketing authorization covers the treatment of adults with moderate-to-severe MASH, the same population covered by Rezdiffra’s accelerated FDA approval last year. Madrigal plans to launch Rezdiffra in Europe on a country-by-country basis, starting with Germany in the fourth quarter of this year.

—Precigen immunotherapy Papzimeos became the first FDA-approved drug for recurrent respiratory papillomatosis. The approval comes with the weight of blockbuster expectations, but the clock is ticking for Precigen to monetize the product. Precigen’s financial reports state the biotech’s cash reserves will last less than one year.

—FDA approval of Tonix Pharmaceuticals’ Tonmya makes it the first new fibromyalgia drug in more than 15 years. The main pharmaceutical ingredient in Tonmya is the old muscle relaxant cyclobenzaprine, which Tonix formulated as a sublingual tablet for rapid absorption into the bloodstream. The company expects its new product will launch in the fourth quarter of this year.

—Ogsiveo, a drug that SpringWorks Therapeutics developed to treat desmoid tumors, now has marketing authorization from the European Commission, making it the first treatment approved in Europe for this rare type of tumor affecting connective tissues. The FDA approved Ogsiveo in 2023. Earlier this year, Merck KGaA acquired SpringWorks earlier this year in a $3.9 billion deal.

—Teva Pharmaceutical’s episodic migraine prevention drug Ajovy expanded its label to include children age 6 to 17, making it the first drug in its class approved for children. Ajovy’s initial approval in 2018 covered its use in adults. Ajovy is an injectable antibody drug designed to block CGRP, a protein associated with migraines.

—Novartis’s cholesterol-lowering drug Leqvio is now approved for first-line use. When the small interfering RNA therapy was initially approved in 2021, the FDA decision covered use of the twice-a-year injection as an adjunct to statin therapy. The latest approval permits use of Leqvio without a statin as an adjunct to diet and exercise for lowering LDL cholesterol in patients who have hypercholesterolemia.

—A label expansion for Novo Nordisk’s hemophilia drug Alhemo makes it more competitive with a Sanofi drug. Alhemo may now be used for preventing bleeding episodes in hemophilia A or B who do not have inhibitors, which are antibodies that can develop that making controlling bleeding more difficult. The initial FDA approval of the drug last year covered its use in hemophilia A or B patients with inhibitors. Sanofi’s fitusiran, brand name Qfitlia, won FDA approval in March as a treatment for hemophilia A or B with or without inhibitors.

—Skytrofa, an Ascendis Pharma drug for growth hormone deficiency, is now approved for adults. The once-weekly injectable drug was initially approved by the FDA in 2021 as a treatment for pediatric growth hormone deficiency.

—Apellis Pharmaceuticals drug Empaveli expanded its label to include treatment of two rare kidney diseases: C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). The infused drug, a complement inhibitor, was initially approved for treating the blood disorder paroxysmal nocturnal hemoglobinuria. A formulation developed as an eye injection is approved for treating geographic atrophy, marketed in this indication under the brand name Syfovre.

—LEO Pharma’s Anzupgo landed FDA approval for the treatment of chronic hand eczema. Applied twice-daily, the topical cream delivers a small molecule that inhibits four signaling proteins driving inflammation responses. Denmark-based LEO already has a presence in eczema, also called atopic dermatitis, with Adbry, an injectable antibody drug that won FDA approval in 2022.

—Blenrep, an antibody drug conjugate for multiple myeloma that GSK withdrew from the market in 2022, is returning with the regulatory blessing of the European Union. The drug is now approved as a second-line multiple myeloma treatment for use in combination with the standard multiple myeloma drug regimen known as BorDex. It’s a comeback for the drug, which GSK voluntarily pulled from the market after it failed its confirmatory Phase 3 study as a monotherapy. A new Phase 3 program evaluated the drug as an earlier line of treatment and in combination with existing multiple myeloma treatments.

GSK will need to wait a little longer for the FDA’s verdict on Blenrep. In July, the U.S. regulator notified the company it is extending the review by three months to allow enough time to review additional information submitted in support of the drug’s application. The new target date for an FDA decision is Oct. 23.

—European authorities awarded a CE Mark for Elecsys pTau181, a Roche blood test for Alzheimer’s disease. This non-invasive test measures levels of phosphorylated Tau 181 protein, which is an indicator of the amyloid protein pathology that is a hallmark of Alzheimer’s. This test, which was developed under a collaboration with Eli Lilly, can be used with other clinical information to rule out Alzheimer’s disease as the cause of cognitive decline.

—The European Commission approved Roche drug inavolisib, brand name Itovebi, as a treatment for PIK3CA-mutated, estrogen ER-positive, HER2-negative locally advanced or metastatic breast cancer following recurrence or within 12 months of completing adjuvant endocrine treatment. This approval covers use of Itovebi in combination with the cancer drugs Ibrance and Faslodex. Itovebi won FDA approval in this indication last October.

—The European Union granted marketing authorization for Aucatzyl, an Autolus Therapeutics CAR T-therapy for advanced cases of B cell acute lymphoblastic leukemia (ALL). This drug is engineered with technology intended to improve its safety compared to other cell therapies. The FDA approved Aucatzyl for relapsed and refractory ALL last November.

—PTC Therapeutics drug sepiapterin, brand name Sephience, received FDA approval for the treatment of the rare metabolic disease phenylketonuria in adults and children age 1 month and older. Results from Phase 3 testing showed the once-daily oral drug reduced blood levels of phenylalanine, an amino acid that builds up in this rare disease. The European Commission approved Sephience in June.

…and Rejections

—PTC Therapeutics did receive some unwelcome regulatory news. The company’s vatiquinone was rejected by the FDA as a treatment for the neuromuscular disorder Friedreich’s ataxia. According to PTC, the complete response letter said clinical data failed to demonstrate substantial evidence of efficacy and another well-controlled study is needed for a resubmission. PTC had submitted the application despite vatiquinone’s Phase 3 failure.

—Regeneron Pharmaceuticals received a complete response letter for odronextamab, a bispecific antibody developed to treat follicular lymphoma. The company said the letter was due to FDA observations at an Indiana manufacturing site that is operated by contract manufacturer Catalent, which was acquired last year by Novo Nordisk. The manufacturing issues have also led the FDA to extend the reviews of applications seeking to expand the label of Eylea to macular edema following retinal vein occlusion and to broaden the dosing schedule to include monthly dosing across all of the eye drug’s approved indications.

—Replimune received a complete response letter for RP1, an oncolytic virus developed as a treatment for advanced melanoma. According to the Woburn, Massachusetts-based biotech, the agency said the pivotal study was not adequate and well controlled and provided insufficient evidence of efficacy. No safety issues were raised, but the FDA’s conclusions about efficacy suggest Replimune may need to run another clinical trial.

Clinical Holds Imposed and Lifted, and a License Suspended

—Rocket Pharmaceuticals has FDA clearance to resume a pivotal Phase 2 test of RP-A501, a gene therapy for the inherited metabolic disorder Danon disease. The regulator placed a clinical hold on the trial in May following a patient death associated with a C3 complement inhibitor that was part of an pretreatment immunosuppression regimen for the gene therapy. Rocket said it will proceed with a lower dose of RP-A501. Pretreatment immunosuppression will continue, but without the C3 inhibitor. If the therapy leads to activation of the complement system, the revised clinical trial protocol specifies a lower threshold for administering the C5 inhibitor Soliris.

—The FDA placed a clinical hold on BiomX’s Phase 2b test of BX004, an experimental phage therapy for cystic fibrosis. According to the company, the regulator’s concerns focus on the third-party nebulizer used to administer BX004. BiomX said it has submitted additional information requested by the FDA.

—The FDA suspended the license of Ixchiq, Valneva’s vaccine for chikungunya, move that came weeks after the agency lifted its pause on dosing of the product. The pause followed reports of serious adverse events including fatalities in elderly vaccine recipients. An FDA risk-benefit assessment led to updated warnings on the label and the lift of the dosing pause earlier this month. But four additional serious adverse events were reported recently outside of the U.S., leading the FDA to suspend Ixchiq’s license.

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