A Phase III, Multicenter, Randomized, Placebo Controlled, Double-blind Study to Assess Efficacy and Safety of Crizanlizumab (5 mg/kg) Versus Placebo, With or Without Hydroxyurea/Hydroxycarbamide Therapy, in Adolescent and Adult Sickle Cell Disease Patients With Frequent Vaso-Occlusive Crises

A phase III, multi-center, randomized, placebo-controlled, double-blind study to assess efficacy and safety of crizanlizumab (5 mg/kg) versus placebo, with or without hydroxyurea/hydroxycarbamide therapy, in adolescent and adult Sickle Cell Disease patients with frequent vaso-occlusive crises.

Start Date24 Oct 2024

Sponsor / Collaborator

Novartis Pharmaceuticals Corp.

NCT05469828 / Not yet recruitingPhase 1/2IIT

Crizanlizumab Improves Tissue Oxygen Supply Demand Matching in Patients With Sickle Cell Anemia

Hypothesis
Efficient unloading of oxygen to regions of high metabolic demand requires a healthy microvasculature to sense local oxygen tension and regulate flow, accordingly. In sickle cell disease patients, the investigators have demonstrated oxygen supply-demand mismatch, or SDM, in proportion to anemia severity. SDM occurs in both the peripheral circulation and the brain, and four characteristics: 1) Hyperemia beyond expected for the level of anemia, 2) Corresponding loss of vascular dilatory reserve, 3) Impaired oxygen unloading to the tissues, and 4) Tissue hypoxia. In sickle cell disease, red blood cell (RBC) and white blood cell (WBC) adhere to vascular endothelium triggering transient or irreversible microvascular damage as well as releasing vasoactive substances that contribute to microvascular dysregulation. The investigators postulate that ongoing microvascular damage/dysregulation in the setting of increased total blood flow contributes to SDM. The investigators believe SEG101, by lowering RBC and WBC adhesion to the microvasculature, will improve SDM and tissue oxygenation.
Objectives
Primary - The investigators will test whether SEG101 improves SDM in patients with sickle cell anemia by measuring the change in tissue oxygenation measured by near infrared spectroscopy (NIRS).
Secondary/Exploratory - The investigators will identify end-organ disease and whether improvement of SDM by SEG101 occurs in patients with sickle cell anemia.

Start Date01 Jul 2024

Sponsor / Collaborator

Children's Hospital Los Angeles

[+1]

NCT05020873 / TerminatedNot Applicable

Prospective, Observational Study in Sickle Cell Disease Patients on Crizanlizumab Treatment in Middle East Countries and India (SPOTLIGHT)

This is a multicenter, prospective, single-arm observational non-interventional study (NIS), which will be conducted in various countries in the Middle East and India.

Start Date04 Nov 2021

Sponsor / Collaborator

Novartis Pharmaceuticals Corp.

100 Clinical Results associated with Crizanlizumab-TMCA

100 Translational Medicine associated with Crizanlizumab-TMCA

100 Patents (Medical) associated with Crizanlizumab-TMCA

102

Literatures (Medical) associated with Crizanlizumab-TMCA

01 Oct 2024·JOURNAL OF PHARMACY TECHNOLOGY

Gene Therapies for Sickle Cell Disease

Review

Author: Singh, Divita ; Weaver, Salome Bwayo ; Wilson, Kierra M.

Background: Sickle cell disease (SCD) is a prevalent autosomal recessive hemoglobinopathy affecting millions worldwide, particularly individuals of African ancestry. Sickle cell disease is a lifelong condition associated with a negative impact on quality of life and mortality, causing complications such as painful vaso-occlusive episodes, acute chest syndrome, stroke, long-term anemia, and end-organ damage. Currently, there are 4 U.S. Food and Drug Administration (FDA)-approved drugs, including hydroxyurea, l-glutamine, voxelotor, and crizanlizumab, which work to alleviate symptoms and prevent complications associated with SCD, albeit without addressing the underlying cause of SCD. Allogeneic hematopoietic stem cell transplant (HSCT) has shown promise as a curative approach to SCD but is limited by donor availability and associated complications. This paper aims to review the efficacy and safety of exagamglogene autotemcel and lovotibeglogene autotemcel for managing patients with SCD, including their place in therapy, cost, and accessibility in clinical care. Data Sources: The authors searched PubMed and Medline from 2017 to 2024, for primary literature on both exagamglogene autotemcel and lovotibeglogene autotemcel. Results: The authors identified relevant studies and summarized the data on the two gene therapies. Conclusion: Exagamglogene autotemcel and lovotibeglogene autotemcel are two management strategies that address the underlying cause of SCD and provide curative potential for patients with SCD.

01 Aug 2024·CLINICAL DRUG INVESTIGATION

Budget Impact of Disease-Modifying Treatments and a CRISPR Gene-Edited Therapy for Sickle Cell Disease

Article

Author: Huys, Isabelle ; Simoens, Steven ; Claes, Kathleen J ; Abdallah, Khadidja

BACKGROUND AND OBJECTIVE:

Treatment of sickle cell disease (SCD) has traditionally focused on symptomatic and preventative care. Recent advances in novel therapeutic developments, likely to be orphan-designated, are anticipated to carry a substantial price tag. This study assesses the potential budget impact of adopting disease-modifying treatments, crizanlizumab and voxelotor, and pioneering CRISPR gene-edited therapy, CTX001, in the Belgian healthcare system.

METHODS:

The perspective of the Belgian healthcare payer (RIZIV-INAMI including patient copayments), a 5-year horizon with a 2-10% uptake of disease-modifying interventions, and a 2% uptake of CTX001 were considered. Data, encompassing target population, current (chronic and acute management, curative hematopoietic stem cell transplantation) and new (crizanlizumab, voxelotor, and CTX001) interventions, clinical effectiveness, adverse events, healthcare resource utilization, and associated costs, were gathered through a comprehensive literature review (first phase) and two Delphi panels involving hematologists (second phase). The cost difference between a "world with and without crizanlizumab, voxelotor, and CTX001" was calculated to obtain the budget impact. Three scenario analyses were conducted: a 5-13% and 4% uptake analysis, a 10-18% and 8% uptake analysis, respectively for disease-modifying treatments (crizanlizumab and voxelotor) and CTX001, and a 0% crizanlizumab uptake and managed entry agreements analysis . A ± 20% univariate sensitivity analysis was performed to test the robustness of the analysis.

RESULTS:

The total five-year cumulative budget impact was estimated at €30,024,968, with 91% attributed to drug acquisition costs. The largest budget impact share was for CTX001 (€25,575,150), while crizanlizumab (€2,301,095) and voxelotor (€2,148,723) was relatively small. In scenarios one and three, a two-fold increase of the cumulative budget impact to €60,731,772 and a four-fold increase to €120,846,256 from the base case was observed. In scenario three, this budget impact decreased by 63% to €11,212,766. Patient population size, number of treated patients, and drug costs influenced the analysis the most, while discontinuation, acute crisis, and adverse event rates had comparatively minimal impact.

CONCLUSIONS:

Belgian decision-makers may consider alternative financing models, such as outcome-based risk-sharing agreements or annuities, to ensure sustainable coverage of these treatments. This study adheres to recommended practices for assessing budget impact of orphan drugs, distinguishing it from earlier studies with potentially weaker methodologies.

01 Jul 2024·JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY

Clinical Practice Patterns in Sickle Cell Disease Treatment: Disease-modifying and Potentially Curative Therapies

Article

Author: Sánchez, Luisanna M ; Fasipe, Titilope A ; Morrone, Kerry ; Frei-Jones, Melissa

Therapeutic options for sickle cell disease (SCD) have increased recently as well as the development of updated national guidelines. It is not known how these options are being offered or to what degree guidelines are incorporated into clinical practice. This study aimed to describe practice patterns for pediatric hematologists regarding the use of disease-modifying and potentially curative therapies for SCD. A 9-section, cross-sectional electronic survey was disseminated during a 3-month period via SurveyMonkey, to members of the American Society of Pediatric Hematology/Oncology Hemoglobinopathy Special Interest Group (ASPHO HSIG). A total of 88 physician members of the ASPHO HSIG were surveyed. Ninety percent of respondents (72/80) start hydroxyurea routinely in patients with HbSS and HbSβ0thalassemia, regardless of disease severity. Laboratory monitoring was recommended every 3 months for stable dosing in 63.8% (51/80). New therapies were recommended for patients on hydroxyurea who were still experiencing SCD complications: L-glutamine 68.5% (37/54) or crizanlizumab 93.1% (54/58). Voxelotor was recommended for patients on hydroxyurea with low hemoglobin in 65.1% (43/66) of cases. Matched sibling transplant was considered for any disease severity by 55.1% (38/69). Gene therapy trials are offered on-site by 29% (20/69). Our study demonstrated the enhanced utilization of hydroxyurea while revealing the unexplored potential of other disease-modifying therapies in SCD. These findings underscore the importance of continued knowledge acquisition about the long-term efficacy of new medical therapies and addressing barriers to the use of proven therapies and guide the development of future studies of optimal SCD management.

News (Medical) associated with Crizanlizumab-TMCA

21 Oct 2024

·www.biospace.com

5 Sickle Cell Therapies to Watch Following Pfizer’s Oxbryta Exit

iStock/ YuLi4ka In the wake of Pfizer’s voluntary market withdrawal of the popular sickle cell disease therapy, BioSpace looks at five investigational drugs currently making their way through the pipeline. A recent fumble has left the sickle cell disease community reeling . Last month, Pfizer abruptly pulled Oxbryta from all global markets after new data showed a higher risk of deaths and complications in treated patients. “[Oxbryta] was one of four medications which we would describe as disease-modifying,” Clifford Takemoto, director of clinical hematology at St. Jude Children’s Research Hospital, told BioSpace . Since the approval of Bristol Myers Squibb’s hydroxyurea for sickle cell disease (SCD) in 1998, only three disease-modifying medications have entered the market, he said. Oxbryta, which won FDA accelerated approval in November 2019, was one of these. Another, Novartis’ Adakveo, has also been called into question after failing to reduce vaso-occlusive crises (VOC) in people with SCD in a Phase III trial last year. EU regulators formally revoked Adakveo’s approval in August 2023, but it remains available in the U.S. “We were optimistic that things were changing,” Takemoto said. “People had hoped that these medications would make a difference.” SCD affects more than 100,000 people in the U.S. and 8 million worldwide. With so few treatment options, it remains a seriously underserved population . Takemoto said the Oxbryta withdrawal has left some patients a “bit angry” and distrustful of new medications, and the question for the community remains: “What’s next?” For Takemoto, the ultimate hope for SCD treatment lies in next-gen transplantation and gene therapy. With the approval of bluebird bio’s Lyfgenia and Vertex and CRISPR Therapeutics’ Casgevy, the potential for gene therapies in SCD is there, even if the rollout is slow and for most, not very accessible, he said. St. Jude is developing its own gene therapy, which the research hospital hopes to move into clinical trials sometime next year. Takemoto also pointed to work being done in bone marrow transplantation intended to reduce toxicity and improve success rates. St. Jude is working on a process that will utilize a “half match,” where a parent’s bone marrow can be used instead of a sibling. These treatments could be transformative, Takemoto said. Here, BioSpace looks at five investigational therapies making their way through the SCD pipeline. Pfizer’s Inclacumab and Osivelotor Oxbryta is not the only SCD asset Pfizer brought onboard with its 2022 acquisition of Global Blood Therapeutics . Two other investigational therapies, inclacumab and osivelotor, were also part of the deal. Inclacumab, a P-selectin inhibitor, is currently being assessed in a Phase III trial to determine its safety and efficacy in reducing VOC. The drug works by binding to P-selectin, a cell adhesion molecule found on platelets. According to Pfizer, this prevents platelets from aggregating, which in turn, is expected to help maintain normal blood flow. Like Oxbryta, osivelotor is a sickle hemoglobin polymerization inhibitor, but with a next-generation design. The asset is currently being studied in a Phase II/III study slated for completion in 2028. While Pfizer has not stated whether the recent findings related to Oxbryta will impact the development of osivelotor, Leerink Partners noted that a Phase II study raised safety concerns, with treatment-emergent adverse events seen in 8 out of 35 patients. Agios Pharmaceuticals’ Mitapivat Agios Pharmaceuticals is developing mitapivat, a pyruvate kinase (PK) activator that is already approved for the treatment of hemolytic anemia in adults with PK deficiency, for SCD. Red blood cells require the PK enzyme for energy, Takemoto explained. In December 2023, the company presented positive data from the Phase II portion of its pivotal RISE UP study. The annualized rate of sickle cell pain crises for patients in the higher dose treatment arm was 0.51 compared to 1.71 for participants in the placebo arm. Takemoto said the oral medication seems to benefit red blood cell health in general and noted that the data look favorable for patients with SCD and other hemolytic anemias. But not everyone is as convinced. Leerink Partners analysts wrote last month of concerns that similar physiological effects could be seen with both Oxbryta and mitapivat. A Phase III readout is expected next year, and the company is targeting a label expansion in 2026. Novo Nordisk’s Etavopivat In 2022, Novo Nordisk paid $1.1 billion to acquire Forma Therapeutics, gaining a lead SCD asset, etavopivat, in the deal. Like mitapivat, etavopivat is also a PK activator, but Novo’s candidate comes with a potential advantage —once daily dosing, versus mitapivat’s twice daily regimen. Etavopivat is currently being studied in a Phase III trial slated for completion in 2026. Phase I data presented by Forma at the American Society of Hematology’s annual meeting in 2021 showed a “sustained increase in hemoglobin and improvement in biomarkers of hemolysis and red blood cell health.” The results, from 15 patients dosed for up to 12 weeks, also showed a trend toward decreasing VOC compared to the 12 months prior to their entering the trial. Takemoto said the candidate “looks very promising” so far but acknowledged that “there are things we don’t know about it. We may learn new side effects as it’s used.” St. Jude will serve as a site for the Phase III trial. Fulcrum Therapeutics’ Pociredir Fulcrum Therapeutics ’ pociredir, a polycomb repressive complex 2 (PRC2) inhibitor, is designed to induce expression of fetal hemoglobin to compensate for the mutated hemoglobin that occurs in SCD. The development path for pociredir has not been without bumps. In February 2023, the FDA slapped Fulcrum with a clinical hold based on previously submitted preclinical data and non-clinical and clinical evidence of hematological malignancies observed with other PRC2 inhibitors. The hold was released six months later. The small molecule, which is currently being studied in a Phase Ib trial, has been well-tolerated in people with SCD with no treatment-related adverse events reported after up to three months exposure, according to a press release announcing the lifted clinical hold.

Clinical ResultPhase 3Phase 2Drug ApprovalPhase 1

26 Sep 2024

·www.outsourcing-pharma.com

Pfizer withdraws sickle cell disease drug Oxbryta amid safety concerns

The withdrawal decision was prompted by clinical data indicating that the overall benefit of Oxbryta “no longer outweighs the risk” in its target patient population, the company said in a public release . Specifically, the trials outline an “imbalance” in vaso-occlusive crises and fatal events that the company will assess further. The move to stop clinical trials of the drug will last while Pfizer further reviews the available data and investigates the findings. Sickle cell disease is a genetic blood disorder where patients have a mutation in hemoglobin, a protein found in red blood cells. This leads the protein to clump together, causing cells to have a sickle shape that can block blood vessels and carry less oxygen, leading to acute pain and anemia. Oxbryta, whose generic name is voxelotor, is an oral small molecule drug that was first approved by the US Food and Drug Administration (FDA) for the treatment of sickle cell disease in 2019, with an EU approval granted in 2022. The drug is designed to make hemoglobin better at carrying oxygen and less prone to clumping together, making red blood cells more functional. The treatment was previously developed by the U.S. firm Global Blood Therapeutics before the company was snapped up by Pfizer for $5.4 billion in 2022 , and bagged Pfizer $328 million in revenues in 2023 . The big pharma company has notified regulators about the data and doesn’t expect the event to impact its full-year 2024 financial guidance. Nevertheless, the company’s stock price on the New York Stock Exchange slumped by more than 1% at the news yesterday (Wednesday 25th September). googletag.cmd.push(function () { googletag.display('text-ad1'); }); Emphasizing Pfizer’s focus on patient safety, chief medical officer and head of worldwide medical and safety, Aida Habtezion, said in the company release that the Pfizer’s “primary concern is for patients who suffer from SCD, which remains a very serious and difficult-to-treat disease with limited treatment options. We advise patients to contact their physicians to discuss alternative treatment while we continue to investigate the findings from our review of the data.” The global market for sickle cell disease was worth $2.25 billion in 2022 and is expected to grow by 20.1% per year to $9.84 billion by 2030. The main players in the market have included Pfizer with Oxbryta, Novartis with the antibody Adakveo (crizanlizumab) and Generex Biotechnology’s Emmaus Medical with Endari (L-glutamine). In 2017, Endari became the first branded drug for sickle cell disease in decades when it was greenlit by the U.S. FDA. Another driver for the sickle cell disease market is gene therapy, with the first cell-based gene therapies – Casgevy by Vertex Pharmaceuticals and Lyfgenia by Bluebird Bio – approved by the U.S. FDA in late 2023 .

Drug ApprovalGene Therapy

10 Mar 2024

·medcitynews.com

Blockbuster BeiGene Cancer Drug Adds Another FDA Approval

A BeiGene drug already used to treat several types of blood cancer now has an additional FDA approval as a treatment for follicular lymphoma. The regulatory decision makes the BeiGene drug the first in its class for treating this type of cancer. The FDA last Thursday approved zanubrutinib, brand name Brukinsa, for patients whose follicular lymphoma has not responded to two or more lines of therapy. BeiGene’s twice-daily capsule is administered alongside periodic infusions of obinutuzumab, brand name Gazyva, a targeted therapy from Roche that is a standard follicular lymphoma treatment. The new approval comes four months after European Commission approval of Brukinsa for follicular lymphoma. Follicular lymphoma is a slow-growing form of non-Hodgkin lymphoma, a cancer affecting white blood cells called lymphocytes. Brukinsa belongs to a class of therapies called BTK inhibitors. The small molecule drug is designed to block Bruton’s tyrosine kinase (BTK), an enzyme that drives cancer cell growth. The approval in follicular lymphoma is based on the results of a clinical trial that enrolled 217 adults and compared treatment with Brukinsa and Gazyva to treatment with Gazyva alone. The main goal was to measure the overall response rate and the duration of response. Results showed an overall response rate of 68% in the study drug arm compared to 46% in the control group. With a median follow-up of 19 months, the median duration of response was not reached in the Brukinsa/Gazyva arm, and was 14 months in the Gazyva group. Across the drug’s clinical trials, the most common adverse reactions included low levels of a type of white blood cell called a neutrophil, upper respiratory tract infections, bleeding, and muscle pain. Brukinsa won its first FDA approval in 2019 for the treatment of mantle cell lymphoma. Its other approved indications are Waldenstrom’s macroglobulinemia, marginal zone lymphoma, and chronic lymphocytic leukemia (CLL). The drug is BeiGene’s top product, accounting for $1.3 billion in revenue in 2023, a more than 128% increase over the product’s sales in 2022. In its annual report, BeiGene said Brukinsa’s U.S. sales growth in 2023 was driven by the product’s launch in CLL. Brukinsa is currently approved in 70 countries. With Brukinsa’s latest FDA nod, the BTK inhibitor joins a small group of therapies already available as third-line treatments for follicular lymphoma. The CAR T-therapies Kymriah from Novartis and Yescarta from Gilead Sciences are such treatment options. Another choice is Lunsumio, a bispecific T cell engager developed by Roche and approved by the FDA in 2022. The FDA nod for Brukinsa in follicular lymphoma is an accelerated approval that requires a post-marketing study to confirm the therapy’s efficacy. BeiGene said a Phase 3 trial already underway will serve as the confirmatory clinical trial for this indication. “This accelerated approval of Brukinsa represents an important advancement, offering the first and only BTK inhibitor treatment for follicular lymphoma patients in the U.S. who have either not responded to initial therapies or have experienced relapse,” Mehrdad Mobasher, BeiGene’s chief medical officer, hematology, said in a prepared statement. Here’s a recap of other recent regulatory news: —Blockbuster Novo Nordisk drug Wegovy, approved as a treatment for weight management, is expanding its label to include reducing cardiovascular risks in those who are obese or overweight. FDA approval of the additional indication is based on results from a clinical trial showing that patients who treated with Wegovy experienced a significantly lower rate of cardiovascular events compared to those who received a placebo. That study followed more than 17,000 participants for up to five years. —Eli Lilly Alzheimer’s disease drug donanemab won’t receive a first quarter 2024 regulatory decision as expected. The FDA wants to convene an advisory panel to discuss the safety and efficacy of the antibody drug, including the unique trial design that allowed patients to complete their course of treatment upon reaching a prespecified level of amyloid plaque clearance. The meeting date has not yet been set. Lilly said holding an advisory committee for donanemab is consistent with the review of Aduhelm and Leqembi, antibody drugs that previously won approvals in Alzheimer’s disease. —Sandoz received FDA approvals for two biosimilars that are interchangeable with the blockbuster Amgen products Prolia and Xgeva, antibody drugs that block a protein called RANKL to treat osteoporosis and bone problems in cancer patients. The new Sandoz drugs, Jubbonti and Wyost, are the first FDA-approved RANKL inhibitor biosimilars. —Partners Formosa Pharmaceuticals and Aimax Therapeutics received FDA approval for a new formulation of a topical steroid developed for treating inflammation and pain following eye surgery. The drug, clobetasol propionate ophthalmic suspension, is made with Formosa’s nanoparticle formulation technology. The Formosa drug will be commercialized in the U.S. by Eyenovia, a New York company that acquired U.S. rights to the steroid. A mid-year launch is planned. Eyenovia also said it also exploring development of this product, delivered with its Optejet dispenser device, as a potential treatment for dry eye disease. —The FDA approved Johnson & Johnson drug Rybrevant as a first-line treatment for non-small cell lung cancer that carries the rare EGFR exon 20 insertion mutation. The J&J antibody drug initially won accelerated approval in 2021. The new FDA decision converts Rybrevant’s status to full approval. It’s based on the results of a confirmatory Phase 3 study that showed a statistically significant improvement in progression-free survival. —EMD Serono, the healthcare business of German company Merck KGaA, also converted an accelerated approval to a traditional one. The FDA approved the company’s Tepmetko for treating adults with advanced non-small cell lung cancer that has MET exon 14 skipping alterations. Tepmetko received accelerated FDA approval in 2021. —The FDA turned down a Minerva Neurosciences application seeking approval of roluperidone as a treatment for symptoms of schizophrenia. The drug is designed to selectively block certain receptors involved in regulating brain functions, such as mood and anxiety. Among the deficiencies cited: The application included a statistical significance finding from only one clinical trial, which on its own is insufficient to provide substantial evidence of effectiveness, the agency told Minerva. The FDA asked Burlington, Massachusetts-based Minerva to submit data from at least one additional well-controlled clinical trial and also provide more data to show the drug’s safety and efficacy when co-administered with other antipsychotic medications. Minerva said it would request a meeting to discuss the FDA letter. —Allecra Therapeutics received FDA approval for Exblifep for treating complicated urinary tract infections in adults. The antibiotic is part of a class of drugs that work by inhibiting synthesis of a bacterium’s cell wall. With the approval, France and Germany-based Allecra receives five-year marketing exclusivity for the product under the Generating Antibiotic Incentives Now Act, which was enacted to incentivize anti-infective product R&D. Allecra said it is in discussions with potential U.S. commercialization partners for Exblifep. —Novartis is withdrawing Adakveo from the U.K. market after post-marketing testing failed to confirm the drug’s benefit. Adakveo was developed to treat vaso-occlusive crises, a complication of sickle cell disease. The exit from the U.K. market follows the withdrawal of the drug from the European Union last year. At that time, the company said it also planned to discuss the study results with the FDA. —The European Commission granted marketing authorization for Pfizer drug Velsipity as a treatment for moderately to severely active ulcerative colitis in patients 16 and older. The once-daily oral drug is a small molecule designed to selectively target and block three S1P receptors. European approval of Velsipity follows the drug’s FDA approval last October. —Iovance Biotherapeutics’ Amtagvi won the first FDA approval for a type of cell therapy called a tumor-infiltrating lymphocyte, or TIL. The regulatory decision also marks the first approval of a cell therapy for treating solid tumors. Amtavi’s approval specifically covers the treatment of advanced cases of melanoma. The one-time treatment carries a $515,000 wholesale price. —Xolair, an old Roche drug whose approved uses include treating asthma, received a new FDA approval for reducing allergic reactions in people who have food allergies. Xolair is an antibody that blocks immunoglobulin E, an antibody produced by the immune system in response to an allergen. The drug’s latest regulatory decision covers adults as well as children age 1 and older who have food allergies. Xolair’s is not an emergency treatment for allergic reactions to food. People who have food allergies should continue to avoid trigger foods. But Xolair reduces allergic reactions in instances of accidental exposure to a food allergen.

Clinical ResultPhase 3Drug ApprovalAccelerated ApprovalImmunotherapy

100 Deals associated with Crizanlizumab-TMCA

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KEGG	Wiki	ATC	Drug Bank
-	Crizanlizumab-TMCA	B06AX01	DB15271

R&D Status

Approved

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Indication	Country/Location	Organization	Date
Anemia, Sickle Cell	US	Novartis Pharmaceuticals Corp.	15 Nov 2019
Vaso-occlusive crisis	US	Novartis Pharmaceuticals Corp.	15 Nov 2019

Developing

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Indication	Highest Phase	Country/Location	Organization	Date
COVID-19	Phase 2	US	Novartis AG	09 Jul 2020
Chronic Kidney Diseases	Phase 2	US	Novartis Pharmaceuticals Corp.	10 Dec 2019
Chronic Kidney Diseases	Phase 2	BR	Novartis Pharmaceuticals Corp.	10 Dec 2019
Chronic Kidney Diseases	Phase 2	FR	Novartis Pharmaceuticals Corp.	10 Dec 2019
Chronic Kidney Diseases	Phase 2	GR	Novartis Pharmaceuticals Corp.	10 Dec 2019
Chronic Kidney Diseases	Phase 2	IE	Novartis Pharmaceuticals Corp.	10 Dec 2019
Chronic Kidney Diseases	Phase 2	LB	Novartis Pharmaceuticals Corp.	10 Dec 2019
Chronic Kidney Diseases	Phase 2	NL	Novartis Pharmaceuticals Corp.	10 Dec 2019
Chronic Kidney Diseases	Phase 2	PA	Novartis Pharmaceuticals Corp.	10 Dec 2019
Chronic Kidney Diseases	Phase 2	ES	Novartis Pharmaceuticals Corp.	10 Dec 2019

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03264989 (SOLACE-adults, CTgov)	Phase 2	Vaso-occlusive crisis \| Anemia, Sickle Cell	57	Crizanlizumab (Crizanlizumab 5.0 mg/kg)	iqqspotpuk(bmxxdkthxq) = nnadwozjdt melqgmnweb (lzpnlohksj, zojcixenaw - wmrdvzggkr) View more	-	23 Apr 2024
				Crizanlizumab (Crizanlizumab 7.5 mg/kg)	nxsnkzatak(cicyqpstbh) = kckgmbocsy ebgwnazfcw (wafswkrvsi, sjabfctwxm - rehbygfvsz) View more
NCT03814746 (STAND, CTgov)	Phase 3	Vaso-occlusive crisis \| Anemia, Sickle Cell	258	Crizanlizumab (SEG101) (Crizanlizumab (SEG101) at 5.0 mg/kg)	fcbuzzwori(mrauegvcen) = vtxfcydfxy ykaghunfob (zjquypblem, bigrwbssqt - ytoqllzeah) View more	-	08 Jan 2024
				Crizanlizumab (SEG101) (Crizanlizumab (SEG101) at 7.5 mg/kg)	fcbuzzwori(mrauegvcen) = pigitbajxe ykaghunfob (zjquypblem, tbovbrbkov - jdnlrkmkmy) View more
NCT03474965 (SOLACE-KIDS, EHA2023)	Phase 2	Anemia, Sickle Cell	50	Crizanlizumab 5 mg/kg	obqbfntezx(bjwpbsxtew) = nvfnmhajhd kamlualltg (lnevzlmozi ) View more	-	08 Jun 2023
NCT03264989 (Pubmed) Manual	Phase 2	Anemia, Sickle Cell	57	crizanlizumab (5.0 mg/kg)	nosnarnnjy(lforuembsk) = rpabjhgzob ppaiozjasp (hrihlixvol ) View more	Positive	10 Nov 2022
				crizanlizumab (7.5 mg/kg)	nosnarnnjy(lforuembsk) = ukcoosxhpm ppaiozjasp (hrihlixvol ) View more
NCT03720626 (CSEG101A2001M, EHA2022)	Not Applicable	Vaso-occlusive crisis \| Anemia, Sickle Cell	188	Crizanlizumab	abkqwtkpaw(xdmmcqzmgo) = vnornbdijx nmgghpilqb (ruuswnxpmz )	-	12 May 2022
NCT04435184 (CRITICAL, CTgov)	Phase 2	COVID-19	54	Crizanlizumab (Crizanlizumab)	vdcapkbwat(zishqexdfk) = jisthmxcvs kqhiqstuxo (vjmvrmpebc, bhixotxees - mpknpszicg) View more	-	09 Nov 2021
				0.9% saline (Placebo Saline)	vdcapkbwat(zishqexdfk) = ussxjqytge kqhiqstuxo (vjmvrmpebc, qzwackasrw - bzxxdbmwny) View more
NCT01895361 (SUSTAIN, CTgov)	Phase 2	Pain \| Anemia, Sickle Cell	198	SelG1 (High-dose SelG1 (Selg1 5.0 mg/kg))	hplmsfqajt(qjcnbdvvsh) = zqyngglepl asguppyjeu (kkckixlxth, vsxvwflmms - bkpacknaxs) View more	-	31 Jan 2020
				SelG1 (Low-dose SelG1 (Selg1 2.5 mg/kg))	hplmsfqajt(qjcnbdvvsh) = ugyhqgkcbg asguppyjeu (kkckixlxth, dvjhwdqbff - jersqtztfz) View more
NCT01895361 (SUSTAIN, Pubmed \| Br Dent J) Manual	Phase 2	Pain \| Anemia, Sickle Cell	198	Crizanlizumab	ktapvdvdav(bhpeiaywdw) = evvrocrbgs dugjbhegxo (efeoocwmkz ) View more	Positive	02 Feb 2017
				Placebo	ktapvdvdav(bhpeiaywdw) = nezziakmkm dugjbhegxo (efeoocwmkz ) View more

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Understand key drug designations in just a few clicks with Synapse.

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Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis