Market Research Report
Graft versus host disease (GVHD)- Market Insight, Epidemiology and Market Forecast -2030
|Graft versus host disease (GVHD)- Market Insight, Epidemiology and Market Forecast -2030|
DelveInsight Business Research LLP
Content info: 429 Pages
Delivery time: 2-10 business days
DelveInsight's 'Graft-versus-host disease (GvHD) - Market Insights, Epidemiology, and Market Forecast-2030' report delivers an in-depth understanding of the GvHD, historical and forecasted epidemiology as well as the GvHD market trends in the United States, EU5 (Germany, France, Italy, Spain, and United Kingdom), and Japan.
The GvHD market report provides current treatment practices, emerging drugs, GvHD market share of the individual therapies, current and forecasted GvHD market size from 2018 to 2030 segmented by seven major markets. The Report also covers current GvHD treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.
Study Period: 2018-2030
GvHD is an immune condition that occurs when immune cells (T-cells) after transplant procedures from the donor (known as the graft or graft cells) attack the recipient patient host's tissues (healthy cells); the disease is a side effect that is common after an allogeneic bone marrow transplant (stem cell transplantation).The two main types of GvHD are acute GvHD and chronic GvHD. Other than this, GvHD is classified into other types, namely: classic acute GvHD, persistent, recurrent, late-onset acute GvHD, classic chronic GvHD, and overlap syndrome and de novo chronic GvHD. Patients who get acute GvHD have a 50% chance of developing chronic GvHD. Acute GvHD (aGvHD) might occur once the donor's cells have engrafted in the transplant recipient and have been observed to affect skin, liver, or gastrointestinal tract. Symptoms might appear within 3 months after the transplant.
Acute GvHD is staged and graded (grade 0-IV) by the number and extent of organ involvement (skin, liver, and gastrointestinal). That staging is then used to determine the overall grade of the patient. Usually, patients with grade III-IV acute GvHD have a much worse outcome than those with grade I-II GvHD. The treatment depends on the grade of patient's GvHD. Chronic GvHD (cGvHD) usually develops later than 100 days after the transplant, and it might occur in the skin, liver, mouth, lungs, gastrointestinal tract, neuromuscular system, or genitourinary tract. Its diagnosis is based on manifestations and not timing. The doctor assesses the GvHD by looking at the number of organs involved and severity. Depending on this, they give it one of the following grades: mild, moderate and severe chronic GvHD. Patients who have an increased risk of developing chronic GvHD are: those who have received stem cells/bone marrow from an HLA mismatched related donor or from an HLA matched unrelated donor, use of peripheral blood stem cells (PBSCs), patients who may have already experienced acute GvHD, older transplant recipients and conditioning regimen..
The diagnosis of aGvHD is based wholly on clinical criteria that can be established by biopsy of one of the three target organs. Additionally, laboratory data and/or imaging studies are also useful tests in the diagnostic approach to GvHD. In case of cGvHD, some symptoms might be very ambiguous, which might make the diagnosis possible only after other causes are excluded. Some tests which are useful in diagnosis are biopsy (liver and skin), endoscopy, X-rays and scans, lung function tests and blood tests.
Treatment is grounded on drugs that reduce the body's immune response and reduce T cells' number. The current hallmark of treatment for GvHD is Immunosuppressant medications. These include both corticosteroid drugs and more advanced medications and techniques that decrease the immune response. Calcineurin Inhibitors (CNIs) are considered to be the backbone of GvHD prophylaxis in HSCT, and can be used as single agents and in combinations as well. Cyclosporine or tacrolimus with short-term methotrexate is mainly used for GvHD prophylaxis in most recipients. Apart from this, immunosuppression using tacrolimus or cyclosporine in combination with mycophenolate mofetil, or sirolimus is also common.
The recommended first-line treatment for both acute and chronic GvHD is systemic steroid therapy (methylprednisolone and prednisone) as a monotherapy and in combinations as well. However, around half of those patients receiving the treatment in first line become refractory to steroid therapy. At present, there are no exact consensus around the management of steroid refractory pool. Ruxolitinib (Jakafi) and Ibrutinib (Imbruvica) are the only two United States Food and Drug Administration (USFDA) approved therapies for the treatment of steroid refractory acute and chronic GvHD, respectively.
The disease epidemiology covered in the report provides historical as well as forecasted GVHD epidemiology [segmented as Total Hematopoietic stem-cell transplantation (HSCT), Total Allogeneic Transplant cases, Total GvHD cases by Types (Acute and Chronic), Total Incident cases of aGvHD by Grading and organ Involvement, Total Incident cases of cGvHD by Grading and organ involvement, Total Treated patients of GvHD and Mortality adjusted GvHD treated patients] in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom), and Japan from 2018 to 2030.
Drug chapter segment of the GvHD report encloses the detailed analysis of GvHD developmental stage pipeline drugs. It also helps to understand the GvHD clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.
GvHD Marketed Therapies
Jakafi (Ruxolitinib): Incyte Corporation
Ruxolitinib (INCB018424) is a potent dual JAK1 and JAK2 inhibitor that exhibits low single digit nanomolar biochemical IC50s for both kinases. The USFDA granted an orphan drug designation to Jakafi for the treatment of acute GvHD. Apart from this, in June 2016, the USFDA granted Breakthrough therapy designation for Jakafi in patients with acute GvHD. In May 2019, the USFDA approved Jakafi for the treatment of steroid-refractory GvHD in adult and pediatric patients 12 years and older
Imbruvica (Ibrutinib): Pharmacyclics (Acquired by Abbvie)/ Janssen
Ibrutinib (previously known as PCI-32765) is a small molecule that works by inhibiting a type of enzyme, called a protein kinase that controls the rate at which certain cells multiply. In particular, ibrutinib has been shown to covalently bind to, and ultimately inhibit, the Bruton's tyrosine kinase (BTK). In August 2017, the US FDA approved Imbruvica for the treatment of adult patients with chronic GvHD after failure of one or more lines of systemic therapy.
Temcell (Ryoncil; Remestemcel-L; Prochymal): JCR therapeutics/ Mesoblast/ Osiris Therapeutics
In September 2015, the Pharmaceuticals and Medical Devices Agency (PMDA) approved Temcell as Japan's first allogeneic regenerative medical product which was launched in February 2016. Temcell was developed to treat corticosteroid-refractory acute GvHD following hematopoietic stem cell transplantation (corticosteroid therapy is considered to be the first-line treatment for the disease).
Note: Full and detailed list of emerging therapies will be provided in the final report.
GVHD Emerging Drugs
Obnitix (MC0518): Medac
Obnitix, also called MC0518, is a new special preparation of mesenchymal stromal cells (MSC) produced with the help of an innovative process by Medac. Obnitix is administered via an infusion. Apart from this, Obnitix is being studied in an ongoing Phase III (NCT04629833: IDUNN) trial at multiple locations across Germany, France, Poland, and Spain.
MaaT013: MaaT Pharma
MaaT Pharma is developing MaaT013, which is made up of allogeneic, full-ecosystem pooled biotherapeutic intestinal microbiota manufactured in France, per the GMP requirements. It is an off-the-shelf, standardized, pooled-donor, high-richness microbiome biotherapeutic in enema formulation. Currently, the company plans to start a multicenter open-label Phase III (NCT04769895) trial, which will begin in October 2021 to evaluate the efficacy of MaaT013 as salvage therapy in acute GvHD patients with gastrointestinal involvement, refractory to ruxolitinib.
Leukotac (Inolimomab): ElsaLys Biotech (Mediolanum Farmaceutici Spa)
Inolimomab is ElsaLys Biotech's potential therapy for the treatment of steroid-refractory aGvHD. It is an immunotherapy monoclonal antibody that targets the interleukin-2 receptor (IL-2), a chemical molecule named cytokine that contributes to the development and proliferation of some white blood cells, including T-cells responsible for aGvHD. Currently, this drug is in Phase III (NCT04289103) of clinical development. ElsaLys is working on expanding compassionate use programs for inolimomab in several other countries in Europe.
KD025 (Belumosudil): Kadmon Corporation
KD025 (Belumosudil) is a late-stage product candidate who is a selective oral inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates inflammatory response and pro-fibrotic processes. It has shown potency in the preclinical studies, and as of now, it is being developed in the Phase II clinical trial for the treatment of cGvHD. In March 2021, the USFDA extended the review period for the NDA for belumosudil to treat cGvHD.
CSL 964 Alpha-1 antitrypsin (AAT): CSL Behring
CSL 964 AAT, also known as Zemaira, is an Alpha1-Proteinase Inhibitor (A1 -PI) being developed by CSL Behring to treat steroid-refractory, aGvHD and prevention of aGvHD in high-risk patients receiving an allogeneic HSCT.
Itacitinib: Incyte Corporation
Itacitinib by Incyte Corporation is an orally administered small molecule that inhibits JAK 1. Itacitinib is being evaluated in GRAVITAS-309, a pivotal Phase III trial of itacitinib in patients with steroid-naive chronic GvHD.
MK-7110 (CD24Fc): OncoImmune/Merck (MSD)
MK-7110, formerly known as CD24Fc, was OncoImmune's lead product, which is now acquired by MSD. It is a first-in-class recombinant fusion protein that targets a novel immune pathway checkpoint. This drug has a dual mechanism of action. A pivotal Phase III (NCT04095858) clinical trial for prophylaxis of GvHD has been initiated nationwide.
EQ001 (Itolizumab; Bmab600): Equillium/Biocon
Itolizumab (EQ001; Bmab600) is a first-in-class immune-modulating antibody therapeutic designed to inhibit CD6, in order to reduce the activation and trafficking of pathogenic T cells that release pro-inflammatory cytokines in a range of autoimmune and inflammatory diseases like aGvHD. Currently, Itolizumab is being studied in Phase I/II (NCT03763318: EQUATE) clinical trial. For next phase, the company expects regulatory interaction in mid-2021 and Phase II/III study initiation is expected in second-half of 2021.
Note: Full and detailed list of emerging therapies will be provided in the final report.
The GvHD market size in the 7MM is expected to change during the study period 2018-2030, at a CAGR of 15.9%. According to the estimates, the highest market size of GVHD is accessed in the United States followed by Japan and Germany, in 2020.
In United States, the total market size of GvHD is expected to increase at a CAGR of 17.9% during the study period (2018-2030).
In EU-5, the total market size of GvHD is expected to increase at a CAGR of 15.5% during the study period (2018-2030).
The total market size of GvHD in Japan is estimated to rise at a CAGR of 5.6%, during the study period (2018-2030).
GvHD Pipeline Development Activities
The drugs which are in pipeline include:
able 19 Mortality adjusted GvHD treated patients in the United States (2018-2030)