The Eisai- and Biogen-partnered Alzheimer’s illness drug Leqembi has been obtainable thus far as an intravenous infusion that takes about an hour. Sufferers will quickly have the choice to decide on an injectable model that may be dosed in 15 seconds.
The brand new FDA-approved injectable formulation is just for upkeep dosing; sufferers nonetheless should begin with intravenously infused Leqembi each two weeks together with the usual monitoring that comes with it. However after 18 months, sufferers could change to a brand new weekly injectable formulation of Leqembi for upkeep remedy, based on the approval announcement launched late Friday simply forward of the Labor Day vacation weekend.
The brand new Eisai and Biogen injectable drug will carry the model identify Leqembi Iqlik (pronounced “I click on”). Administered through an auto-injection pen, dosing could also be carried out at residence, easing the burden on each sufferers and their caregivers. Eisai and Biogen are aiming for an Oct. 6 launch of Iqlik.
Whereas IV dosing of Leqembi takes about an hour, set-up time and the required post-infusion commentary interval means sufferers and their caregivers should plan to be at an infusion heart for a number of hours. Sufferers have to be monitored for opposed results of the drug, an antibody that clears amyloid plaques. Bleeding and swelling is a recognized and probably deadly opposed impact of this class of Alzheimer’s medicine. Clinicians monitor for this complication, known as amyloid-related imaging abnormalities with edema (ARIA-E), by MRI scans beginning with the fifth infusion. Final Thursday, the FDA issued a security communication recommending MRI scanning earlier than the third infusion to probably establish ARIA-E even earlier. This modification is now mirrored within the drug’s up to date label.
Iqlik’s FDA submission was based mostly on the outcomes of a Section 3 open-label extension research that evaluated a variety of subcutaneous doses in sufferers with early Alzheimer’s. Knowledge confirmed that transitioning from IV-infused Leqembi to the injectable model of the drug at 18 months maintained scientific and biomarker advantages that had been akin to continued IV dosing. Whereas the protection of the injectable formulation was just like that of IV upkeep remedy, Eisai highlighted one key distinction: systemic reactions had been a lot much less widespread with injectable dosing.
Leqembi and Leqembi Iqlik stem from a multi-drug Alzheimer’s alliance between Eisai and Biogen. The 2 firms share in growth and commercialization of the partnered medicine. Whereas Biogen led growth of the now defunct Alzheimer’s drug Aduhelm, Eisai led growth of Leqembi, which secured full FDA approval in 2023.
Iqlik carries a listing value of $375 per autoinjector, which works out to $19,500 a yr. By comparability, Eisai initially set a $26,500 checklist value for infused Leqembi. In a press release issued about Iqlik’s pricing, Eisai famous that below Medicare Half D at the moment sufferers have a $2,000 cap on their whole annual out-of-pocket spending on lined medicine. Meaning beneficiaries pays not more than $2,000 a yr, and maybe even much less, Eisai stated.
Dr. Howard Fillit, co-founder and chief science officer of the Alzheimer’s Drug Discovery Basis, views subcutaneous Leqembi as essential to creating the drug extra accessible to sufferers, shifting the sector of Alzheimer’s remedy towards the bypassing of infusions altogether.
“This milestone lessens the burden on sufferers and caregivers by decreasing the logistical challenges of receiving Alzheimer’s remedy, whereas additionally bringing us nearer to the day when sufferers can extra simply obtain a mix of medication, probably administered from residence,” Fillit stated in an emailed assertion.
The FDA issued a number of different notable regulatory approvals to shut out August. Right here’s a recap of these selections and different latest regulatory strikes:
Regulatory Approvals…
—Sanofi’s immunology portfolio is including a projected blockbuster vendor with the FDA approval of rilzabrutinib, model identify Wayrilz, for the remedy of adults with power immune thrombocytopenia (ITP), a uncommon immune dysfunction resulting in low platelet ranges. Wayrilz is an oral small molecule inhibitor of an enzyme known as Bruton’s tyrosine kinase (BTK). The FDA choice for the twice-daily capsule makes it the primary BTK inhibitor permitted within the U.S. for ITP. The drug got here to Sanofi as a part of the pharma big’s $3.7 billion acquisition of Principia Biopharma in 2020.
—The FDA permitted up to date messenger RNA Covid-19 vaccines from companions Pfizer and BioNTech in addition to Moderna, however for narrower use than final yr’s pictures. The approval of each vaccines covers adults age 65 and older. For youthful folks — age 5 to 64 for Pfizer and BioNTech’s Comirnaty and age 12 to 64 for Moderna’s Spikevax — the approvals cowl those that have a minimum of one underlying situation putting them at elevated danger of extreme outcomes from a Covid-19 an infection. Beforehand, these vaccines had been permitted for broader populations. The most recent approvals for Comirnaty and Spikevax are consistent with the FDA’s Could approval of Novavax’s protein-based Covid vaccine, Nuvaxovid, which is being marketed by Sanofi below a deal struck final yr.
—Startup ExCellThera obtained conditional advertising and marketing authorization in Europe for Zemcelpro, a twine blood-derived cell remedy for blood cancers. The approval particularly covers sufferers unable to discover a matched donor for a stem cell transplant, a typical remedy for such cancers.
—The European Fee permitted Gilead Sciences’ lenacapavir, a twice-a-year-injection, as a pre-exposure prophylaxis drug for HIV. It will likely be marketed in Europe below the model identify Yeytuo. Within the U.S., the shot is marketed below the model identify Yeztugo following its FDA approval in June. The drug remains to be below regulatory evaluation in a number of extra international locations.
—FDA approval of Ionis Prescription drugs drug Dawnzera offers the biotech entry into the crowded marketplace for the uncommon illness hereditary angioedema. However Ionis goals to face out with scientific knowledge exhibiting sufferers who switched from different medicine achieved even higher discount within the swelling assaults attributable to this uncommon illness.
—The European Fee permitted Madrigal Pharmaceutical’s Rezdiffra for the remedy of metabolic dysfunction-associated steatohepatitis, the fatty liver illness known as MASH. The conditional advertising and marketing authorization covers the remedy of adults with moderate-to-severe MASH, the identical inhabitants lined by Rezdiffra’s accelerated FDA approval final yr. Madrigal plans to launch Rezdiffra in Europe on a country-by-country foundation, beginning with Germany within the fourth quarter of this yr.
—Precigen immunotherapy Papzimeos turned the primary FDA-approved drug for recurrent respiratory papillomatosis. The approval comes with the burden of blockbuster expectations, however the clock is ticking for Precigen to monetize the product. Precigen’s monetary experiences state the biotech’s money reserves will final lower than one yr.
—FDA approval of Tonix Prescription drugs’ Tonmya makes it the primary new fibromyalgia drug in additional than 15 years. The primary pharmaceutical ingredient in Tonmya is the previous muscle relaxant cyclobenzaprine, which Tonix formulated as a sublingual pill for fast absorption into the bloodstream. The corporate expects its new product will launch within the fourth quarter of this yr.
—Ogsiveo, a drug that SpringWorks Therapeutics developed to deal with desmoid tumors, now has advertising and marketing authorization from the European Fee, making it the primary remedy permitted in Europe for this uncommon kind of tumor affecting connective tissues. The FDA permitted Ogsiveo in 2023. Earlier this yr, Merck KGaA acquired SpringWorks in a $3.9 billion deal.
—Teva Pharmaceutical’s episodic migraine prevention drug Ajovy expanded its label to incorporate kids age 6 to 17, making it the primary drug in its class permitted for kids. Ajovy’s preliminary approval in 2018 lined its use in adults. Ajovy is an injectable antibody drug designed to dam CGRP, a protein related to migraines.
—Novartis’s cholesterol-lowering drug Leqvio is now permitted for first-line use. When the small interfering RNA remedy was initially permitted in 2021, the FDA choice lined use of the twice-a-year injection as an adjunct to statin remedy. The most recent approval permits use of Leqvio with out a statin as an adjunct to weight loss plan and train for decreasing LDL ldl cholesterol in sufferers who’ve hypercholesterolemia.
—A label growth for Novo Nordisk’s hemophilia drug Alhemo makes it extra aggressive with a Sanofi drug. Alhemo could now be used for stopping bleeding episodes in hemophilia A or B sufferers who wouldn’t have inhibitors, that are antibodies that may develop that making controlling bleeding harder. The preliminary FDA approval of the drug final yr lined its use in hemophilia A or B sufferers with inhibitors. Sanofi’s fitusiran, model identify Qfitlia, gained FDA approval in March as a remedy for hemophilia A or B with or with out inhibitors.
—Skytrofa, an Ascendis Pharma drug for development hormone deficiency, is now permitted for adults. The once-weekly injectable drug was initially permitted by the FDA in 2021 as a remedy for pediatric development hormone deficiency.
—Apellis Prescription drugs drug Empaveli expanded its label to incorporate remedy of two uncommon kidney illnesses: C3 glomerulopathy (C3G) and first immune advanced membranoproliferative glomerulonephritis (IC-MPGN). The infused drug, a complement inhibitor, was initially permitted for treating the blood dysfunction paroxysmal nocturnal hemoglobinuria. A formulation developed as an eye fixed injection is permitted for treating geographic atrophy, marketed on this indication below the model identify Syfovre.
—LEO Pharma’s Anzupgo landed FDA approval for the remedy of power hand eczema. Utilized twice-daily, the topical cream delivers a small molecule that inhibits 4 signaling proteins driving irritation responses. Denmark-based LEO already has a presence in eczema, additionally known as atopic dermatitis, with Adbry, an injectable antibody drug that gained FDA approval in 2022.
—Blenrep, an antibody drug conjugate for a number of myeloma that GSK withdrew from the market in 2022, is returning with the regulatory blessing of the European Union. The drug is now permitted as a second-line a number of myeloma remedy to be used together with the usual a number of myeloma drug routine often known as BorDex. It’s a comeback for the drug, which GSK voluntarily pulled from the market after it failed its confirmatory Section 3 research as a monotherapy. A brand new Section 3 program evaluated the drug as an earlier line of remedy and together with present a number of myeloma therapies.
GSK might want to wait somewhat longer for the FDA’s verdict on Blenrep. In July, the U.S. regulator notified the corporate it’s extending the evaluation by three months to permit sufficient time to evaluation further info submitted in assist of the drug’s utility. The brand new goal date for an FDA choice is Oct. 23.
—European authorities awarded a CE Mark for Elecsys pTau181, a Roche blood take a look at for Alzheimer’s illness. This non-invasive take a look at measures ranges of phosphorylated Tau 181 protein, which is an indicator of the amyloid protein pathology that may be a hallmark of Alzheimer’s. This take a look at, which was developed below a collaboration with Eli Lilly, can be utilized with different scientific info to rule out Alzheimer’s illness as the reason for cognitive decline.
—The European Fee permitted Roche drug inavolisib, model identify Itovebi, as a remedy for PIK3CA-mutated, estrogen ER-positive, HER2-negative regionally superior or metastatic breast most cancers following recurrence or inside 12 months of finishing adjuvant endocrine remedy. This approval covers use of Itovebi together with the most cancers medicine Ibrance and Faslodex. Itovebi gained FDA approval on this indication final October.
—The European Union granted advertising and marketing authorization for Aucatzyl, an Autolus Therapeutics CAR T-therapy for superior instances of B cell acute lymphoblastic leukemia (ALL). This drug is engineered with know-how supposed to enhance its security in comparison with different cell therapies. The FDA permitted Aucatzyl for relapsed and refractory ALL final November.
—PTC Therapeutics drug sepiapterin, model identify Sephience, obtained FDA approval for the remedy of the uncommon metabolic illness phenylketonuria in adults and kids age 1 month and older. Outcomes from Section 3 testing confirmed the once-daily oral drug diminished blood ranges of phenylalanine, an amino acid that builds up on this uncommon illness. The European Fee permitted Sephience in June.
…and Rejections
—PTC Therapeutics did obtain some unwelcome regulatory information. The corporate’s vatiquinone was rejected by the FDA as a remedy for the neuromuscular dysfunction Friedreich’s ataxia. Based on PTC, the entire response letter stated scientific knowledge did not reveal substantial proof of efficacy and one other well-controlled research is required for a resubmission. PTC had submitted the appliance regardless of vatiquinone’s Section 3 failure.
—Regeneron Prescription drugs obtained an entire response letter for odronextamab, a bispecific antibody developed to deal with follicular lymphoma. The corporate stated the letter was on account of FDA observations at an Indiana manufacturing website that’s operated by contract producer Catalent, which was acquired final yr by Novo Nordisk. The manufacturing points have additionally led the FDA to increase the critiques of functions in search of to increase the label of Eylea to macular edema following retinal vein occlusion and to broaden the dosing schedule to incorporate month-to-month dosing throughout the entire eye drug’s permitted indications.
—Replimune obtained an entire response letter for RP1, an oncolytic virus developed as a remedy for superior melanoma. Based on the Woburn, Massachusetts-based biotech, the company stated the pivotal research was not sufficient and properly managed and supplied inadequate proof of efficacy. No questions of safety had been raised, however the FDA’s conclusions about efficacy counsel Replimune could have to run one other scientific trial.
Scientific Holds Imposed and Lifted, and a License Suspended
—Rocket Prescription drugs has FDA clearance to renew a pivotal Section 2 take a look at of RP-A501, a gene remedy for the inherited metabolic dysfunction Danon illness. The regulator positioned a scientific maintain on the trial in Could following a affected person loss of life related to a C3 complement inhibitor that was a part of an pretreatment immunosuppression routine for the gene remedy. Rocket stated it would proceed with a decrease dose of RP-A501. Pretreatment immunosuppression will proceed, however with out the C3 inhibitor. If the remedy results in activation of the complement system, the revised scientific trial protocol specifies a decrease threshold for administering the C5 inhibitor Soliris.
—The FDA positioned a scientific maintain on BiomX’s Section 2b take a look at of BX004, an experimental phage remedy for cystic fibrosis. Based on the corporate, the regulator’s considerations concentrate on the third-party nebulizer used to manage BX004. BiomX stated it has submitted further info requested by the FDA.
—The FDA suspended the license of Ixchiq, Valneva’s vaccine for chikungunya, transfer that got here weeks after the company lifted its pause on dosing of the product. The pause adopted experiences of great opposed occasions together with fatalities in aged vaccine recipients. An FDA risk-benefit evaluation led to up to date warnings on the label and the elevate of the dosing pause earlier this month. However 4 further severe opposed occasions had been reported not too long ago outdoors of the U.S., main the FDA to droop Ixchiq’s license.
Photograph: Akio Kon/Bloomberg, through Getty Photos
