The Eisai- and Biogen-partnered Alzheimer’s illness drug Leqembi has been accessible to this point 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 might swap to a brand new weekly injectable formulation of Leqembi for upkeep remedy, in response to 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 by way of an auto-injection pen, dosing could also be executed 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 middle for a number of hours. Sufferers should be monitored for adversarial results of the drug, an antibody that clears amyloid plaques. Bleeding and swelling is a recognized and doubtlessly deadly adversarial impact of this class of Alzheimer’s medication. Clinicians monitor for this complication, known as amyloid-related imaging abnormalities with edema (ARIA-E), by way of MRI scans beginning with the fifth infusion. Final Thursday, the FDA issued a security communication recommending MRI scanning earlier than the third infusion to doubtlessly determine 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 Part 3 open-label extension examine that evaluated a spread of subcutaneous doses in sufferers with early Alzheimer’s. Information 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 similar to continued IV dosing. Whereas the security of the injectable formulation was much like that of IV upkeep therapy, 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 medication. 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 an inventory value of $375 per autoinjector, which works out to $19,500 a yr. By comparability, Eisai initially set a $26,500 record value for infused Leqembi. In an announcement issued about Iqlik’s pricing, Eisai famous that beneath Medicare Half D at the moment sufferers have a $2,000 cap on their whole annual out-of-pocket spending on lined medication. Meaning beneficiaries pays not more than $2,000 a yr, and even perhaps much less, Eisai stated.
Dr. Howard Fillit, co-founder and chief science officer of the Alzheimer’s Drug Discovery Basis, views subcutaneous Leqembi as a vital 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 lowering the logistical challenges of receiving Alzheimer’s therapy, whereas additionally bringing us nearer to the day when sufferers can extra simply obtain a mix of medicine, doubtlessly 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 choices 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 therapy of adults with continual 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 determination for the twice-daily tablet makes it the primary BTK inhibitor accredited within the U.S. for ITP. The drug got here to Sanofi as a part of the pharma large’s $3.7 billion acquisition of Principia Biopharma in 2020.
—The FDA accredited 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 at the least one underlying situation inserting them at elevated danger of extreme outcomes from a Covid-19 an infection. Beforehand, these vaccines had been accredited for broader populations. The most recent approvals for Comirnaty and Spikevax are in keeping with the FDA’s Could approval of Novavax’s protein-based Covid vaccine, Nuvaxovid, which is being marketed by Sanofi beneath a deal struck final yr.
—Startup ExCellThera obtained conditional advertising 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, an ordinary therapy for such cancers.
—The European Fee accredited Gilead Sciences lenacapavir, a twice-a-year-injection, as a pre-exposure prophylaxis drug for HIV. It is going to be marketed in Europe beneath the model identify Yeytuo. Within the U.S., the shot is marketed beneath the model identify Yeztugo following its FDA approval in June. The drug continues to be beneath regulatory overview in a number of extra international locations.
—FDA approval of Ionis Prescribed 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 displaying sufferers who switched from different medication achieved even higher discount within the swelling assaults brought on by this uncommon illness.
—The European Fee accredited Madrigal Pharmaceutical’s Rezdiffra for the therapy of metabolic dysfunction-associated steatohepatitis, the fatty liver illness known as MASH. The conditional advertising authorization covers the therapy 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 grew to become the primary FDA-approved drug for recurrent respiratory papillomatosis. The approval comes with the load of blockbuster expectations, however the clock is ticking for Precigen to monetize the product. Precigen’s monetary stories state the biotech’s money reserves will final lower than one yr.
—FDA approval of Tonix Prescribed 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 authorization from the European Fee, making it the primary therapy accredited in Europe for this uncommon kind of tumor affecting connective tissues. The FDA accredited Ogsiveo in 2023. Earlier this yr, Merck KGaA acquired SpringWorks earlier this yr 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 accredited for youngsters. 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 accredited for first-line use. When the small interfering RNA remedy was initially accredited in 2021, the FDA determination lined use of the twice-a-year injection as an adjunct to statin remedy. The most recent approval permits use of Leqvio and not using a statin as an adjunct to food regimen and train for reducing LDL ldl cholesterol in sufferers who’ve hypercholesterolemia.
—A label enlargement for Novo Nordisk’s hemophilia drug Alhemo makes it extra aggressive with a Sanofi drug. Alhemo might now be used for stopping bleeding episodes in hemophilia A or B 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, received FDA approval in March as a therapy for hemophilia A or B with or with out inhibitors.
—Skytrofa, an Ascendis Pharma drug for development hormone deficiency, is now accredited for adults. The once-weekly injectable drug was initially accredited by the FDA in 2021 as a therapy for pediatric development hormone deficiency.
—Apellis Prescribed drugs drug Empaveli expanded its label to incorporate therapy of two uncommon kidney illnesses: C3 glomerulopathy (C3G) and first immune advanced membranoproliferative glomerulonephritis (IC-MPGN). The infused drug, a complement inhibitor, was initially accredited for treating the blood dysfunction paroxysmal nocturnal hemoglobinuria. A formulation developed as a watch injection is accredited for treating geographic atrophy, marketed on this indication beneath the model identify Syfovre.
—LEO Pharma’s Anzupgo landed FDA approval for the therapy of continual 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 received 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 accredited as a second-line a number of myeloma therapy to be used together with the usual a number of myeloma drug routine generally known as BorDex. It’s a comeback for the drug, which GSK voluntarily pulled from the market after it failed its confirmatory Part 3 examine as a monotherapy. A brand new Part 3 program evaluated the drug as an earlier line of therapy and together with current a number of myeloma remedies.
GSK might want to wait a bit longer for the FDA’s verdict on Blenrep. In July, the U.S. regulator notified the corporate it’s extending the overview by three months to permit sufficient time to overview extra info submitted in assist of the drug’s utility. The brand new goal date for an FDA determination 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 beneath 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 accredited Roche drug inavolisib, model identify Itovebi, as a therapy for PIK3CA-mutated, estrogen ER-positive, HER2-negative domestically superior or metastatic breast most cancers following recurrence or inside 12 months of finishing adjuvant endocrine therapy. This approval covers use of Itovebi together with the most cancers medication Ibrance and Faslodex. Itovebi received FDA approval on this indication final October.
—The European Union granted advertising authorization for Aucatzyl, an Autolus Therapeutics CAR T-therapy for superior circumstances of B cell acute lymphoblastic leukemia (ALL). This drug is engineered with know-how meant to enhance its security in comparison with different cell therapies. The FDA accredited Aucatzyl for relapsed and refractory ALL final November.
—PTC Therapeutics drug sepiapterin, model identify Sephience, obtained FDA approval for the therapy of the uncommon metabolic illness phenylketonuria in adults and youngsters age 1 month and older. Outcomes from Part 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 accredited Sephience in June.
…and Rejections
—PTC Therapeutics did obtain some unwelcome regulatory information. The corporate’s vatiquinone was rejected by the FDA as a therapy for the neuromuscular dysfunction Friedreich’s ataxia. In line with PTC, the entire response letter stated scientific knowledge did not show substantial proof of efficacy and one other well-controlled examine is required for a resubmission. PTC had submitted the applying regardless of vatiquinone’s Part 3 failure.
—Regeneron Prescribed drugs obtained a full response letter for odronextamab, a bispecific antibody developed to deal with follicular lymphoma. The corporate stated the letter was because 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 lengthen the critiques of purposes searching for 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 accredited indications.
—Replimune obtained a full response letter for RP1, an oncolytic virus developed as a therapy for superior melanoma. In line with the Woburn, Massachusetts-based biotech, the company stated the pivotal examine was not satisfactory and nicely managed and supplied inadequate proof of efficacy. No issues of safety had been raised, however the FDA’s conclusions about efficacy counsel Replimune might must run one other scientific trial.
Medical Holds Imposed and Lifted, and a License Suspended
—Rocket Prescribed drugs has FDA clearance to resume a pivotal Part 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 demise related to a C3 complement inhibitor that was a part of an pretreatment immunosuppression routine for the gene remedy. Rocket stated it is going to 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 Part 2b take a look at of BX004, an experimental phage remedy for cystic fibrosis. In line with the corporate, the regulator’s considerations concentrate on the third-party nebulizer used to manage BX004. BiomX stated it has submitted extra 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 stories of great adversarial occasions together with fatalities in aged vaccine recipients. An FDA risk-benefit evaluation led to up to date warnings on the label and the raise of the dosing pause earlier this month. However 4 extra critical adversarial occasions had been reported lately outdoors of the U.S., main the FDA to droop Ixchiq’s license.
Picture: Akio Kon/Bloomberg, by way of Getty Photos
