MedPath

FDA Approves RYTELO™ (imetelstat) for Transfusion-Dependent Anemia in Low-Risk MDS

• The FDA has approved RYTELO™ (imetelstat) for adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia. • RYTELO is a first-in-class telomerase inhibitor that binds to the RNA component of human telomerase, inhibiting its activity. • The drug is indicated for patients who have not responded to, lost response to, or are ineligible for erythropoiesis-stimulating agents (ESA). • Biologics by McKesson has been selected as a limited specialty pharmacy provider for RYTELO, ensuring access to this vital therapy.

The U.S. Food and Drug Administration (FDA) has approved RYTELO™ (imetelstat) for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia. This approval marks a significant advancement for patients requiring four or more red blood cell units over eight weeks and who have not responded to, have lost response to, or are ineligible for erythropoiesis-stimulating agents (ESA).
RYTELO, developed by Geron Corporation, is a first-in-class telomerase inhibitor. It functions by binding to the template region of the RNA component of human telomerase (hTR), thereby inhibiting telomerase enzymatic activity and preventing telomere binding. Increased telomerase activity has been observed in MDS malignant stem and progenitor cells, making it a relevant target for therapeutic intervention.

Mechanism of Action

RYTELO's mechanism of action involves the inhibition of telomerase, an enzyme crucial for maintaining the length of telomeres, which are protective caps on the ends of chromosomes. By inhibiting telomerase, imetelstat can lead to telomere shortening in malignant cells, potentially halting their proliferation. This mechanism is particularly relevant in MDS, where increased telomerase activity contributes to the survival and proliferation of malignant stem and progenitor cells.

Clinical Significance

The approval of RYTELO addresses a critical unmet need in the treatment of low-risk MDS patients with transfusion-dependent anemia. These patients often face significant morbidity due to the need for regular blood transfusions, including iron overload and alloimmunization. Current treatment options, such as ESAs, have limited efficacy in many patients, highlighting the importance of new therapeutic approaches.

McKesson's Role

Biologics by McKesson, an independent specialty pharmacy specializing in oncology and rare disease areas, has been selected by Geron Corporation as a limited specialty pharmacy provider for RYTELO. This collaboration aims to ensure that patients have access to this novel therapy. "As a telomerase inhibitor, RYTELO holds great promise in the fight against transfusion-dependent anemia in LR-MDS," said George Paschal, senior director, business development, Biologics by McKesson. "We are proud to collaborate with Geron Corporation to be a specialty pharmacy provider for RYTELO and provide this vital therapy for valued healthcare providers, and ultimately patients in need."
Biologics by McKesson offers a multidisciplinary approach to patient care, including pharmacists with in-depth knowledge of therapies, experienced nurses, and financial counselors who assist patients with financial assistance programs.
Subscribe Icon

Stay Updated with Our Daily Newsletter

Get the latest pharmaceutical insights, research highlights, and industry updates delivered to your inbox every day.

Related Topics

RESTEM's Restem-L Receives FDA Fast Track Designation for Idiopathic Inflammatory Myopathy

RESTEM's Restem-L, an umbilical cord-derived stem cell therapy, has received Fast Track designation from the FDA for Idiopathic Inflammatory Myopathy (IIM).
Phase 1 data showed Restem-L led to a 78% clinical improvement and a 35% reduction in steroid use within six months for IIM patients.

European Commission Approves Imetelstat for Transfusion-Dependent Anemia in Lower-Risk MDS

The European Commission has granted marketing authorization for imetelstat (Rytelo) as the first telomerase inhibitor approved for transfusion-dependent anemia in lower-risk myelodysplastic syndrome patients ineligible for or unresponsive to erythropoietin-based therapy.
In the pivotal phase 3 IMerge trial, imetelstat demonstrated significant efficacy with 40% of patients achieving red blood cell transfusion independence for at least 8 consecutive weeks compared to 15% with placebo (P = .0008).

CHMP Recommends Approval of Geron's Rytelo for Lower-Risk Myelodysplastic Syndromes

The Committee for Medicinal Products for Human Use (CHMP) has recommended Rytelo (imetelstat) for approval in the EU for transfusion-dependent anemia due to lower-risk myelodysplastic syndromes.
Rytelo targets patients who have had an unsatisfactory response to or are ineligible for erythropoietin-based therapies, offering a new treatment option.

FDA Grants Fast Track Designation to Rigel's R289 for Lower-Risk Myelodysplastic Syndrome

The FDA has granted Fast Track designation to R289, Rigel Pharmaceuticals' IRAK1/4 inhibitor, for previously treated transfusion-dependent lower-risk myelodysplastic syndrome (MDS).
R289 is currently being evaluated in a Phase 1b study for safety, tolerability, pharmacokinetics, and preliminary activity in relapsed or refractory lower-risk MDS patients.

FDA Clears Phase 2 Trial of Leronlimab for Microsatellite-Stable Colorectal Cancer

The FDA has cleared CytoDyn's phase 2 trial of leronlimab in relapsed/refractory microsatellite stable colorectal cancer (MSS CRC).
The trial, in partnership with Syneos Health, will begin patient enrollment in early 2025 after a kickoff meeting in November 2024.

CytoDyn Receives FDA Clearance for Phase II Oncology Trial of Leronlimab in Colorectal Cancer

CytoDyn has received FDA clearance to begin a Phase II oncology trial evaluating leronlimab in patients with relapsed/refractory microsatellite stable colorectal cancer.
The trial follows productive feedback sessions with the FDA and submission of a final study protocol in September 2024, marking a positive development in the company's relationship with the agency.

FDA Approves Keytruda for First-Line Treatment of Malignant Pleural Mesothelioma

The FDA has approved Keytruda (pembrolizumab) in combination with pemetrexed and platinum chemotherapy as a first-line treatment for unresectable advanced or metastatic malignant pleural mesothelioma.
The approval was based on the Phase 2/3 KEYNOTE-483 trial, which demonstrated a statistically significant improvement in overall survival compared to chemotherapy alone.

FDA Approves RYTELO™ (imetelstat) for Lower-Risk MDS with Transfusion-Dependent Anemia

The U.S. Food and Drug Administration (FDA) has approved RYTELO™ (imetelstat), a first-in-class telomerase inhibitor, for the treatment of adult patients with lower-risk myelodysplastic syndromes (MDS) who suffer from transfusion-dependent anemia. This approval is based on the results from the IMerge Phase 3 clinical trial, which demonstrated significant benefits in terms of red blood cell transfusion independence and increases in hemoglobin levels among patients.

FDA Approves Geron's RYTELO™ (imetelstat) for Lower-Risk MDS

The FDA has approved Geron's RYTELO™ (imetelstat) for adult patients with lower-risk myelodysplastic syndromes (LR-MDS) who have transfusion-dependent anemia and are unresponsive to or ineligible for erythropoiesis-stimulating agents (ESA).
The approval was based on the IMerge Phase 3 trial, where RYTELO™ significantly increased red blood cell transfusion independence compared to placebo, with nearly 40% achieving independence for at least eight weeks.

Mereo BioPharma Advances Rare Disease Pipeline with Setrusumab and Alvelestat

Mereo BioPharma's partner, Ultragenyx, anticipates completing enrollment for the Phase 3 Orbit study of setrusumab in Osteogenesis Imperfecta (OI) by the end of Q1 2024.
A global Phase 3 study design for alvelestat in Alpha-1 Antitrypsin Deficiency-associated Lung Disease (AATD-LD) has been aligned upon following interactions with the FDA.

Reference News

[1]
RYTELOTM (imetelstat), FDA Approved for Treatment of Patients with Low- to Intermediate-1 ...
mckesson.com · Sep 9, 2024

Biologics by McKesson selected by Geron Corp. as a specialty pharmacy provider for RYTELO™ (imetelstat), a telomerase in...

© Copyright 2025. All Rights Reserved by MedPath
HomeTerms & ConditionsPrivacy Policy