FDA Grants Orphan Drug Exclusivity to EVOMELA™ (melphalan) for Injection, Indicated for Multiple Myeloma
"We are pleased to receive FDA Orphan Drug Exclusivity for EVOMELA, another important regulatory milestone for Spectrum," said Rajesh C. Shrotriya, MD, Chairman and Chief Executive Officer of Spectrum Pharmaceuticals. "EVOMELA is reconstituted with normal saline and does not contain propylene glycol. EVOMELA's formulation is based on Captisol® technology and is stable at room temperature for 1 hour following reconstitution and for an additional 4 hours after further dilution. We believe EVOMELA has the potential to become an important therapy for multiple myeloma patients undergoing high-dose conditioning treatment prior to hematopoietic stem cell transplantation and fits very well with our existing Hematology/Oncology infrastructure. Revenues from our niche products like EVOMELA help us develop our late-stage drugs that target blockbuster markets."
The FDA Orphan Drug Designation program provides a special status to
drugs and biologics intended to treat, diagnose or prevent so-called
orphan diseases and disorders that affect fewer than 200,000 people in
Spectrum Pharmaceuticals gained global development and commercialization
rights to EVOMELA from
About Multiple Myeloma
Multiple Myeloma is a systemic malignancy of plasma cells that accumulate in the bone marrow, usually associated with monoclonal antibody secretion, and results in bone marrow failure and bone destruction. It is the second most common hematologic disease with nearly 30,000 new cases projected in the US in 2016 and over 11,000 deaths annually (American Cancer Society Stats, 2016). The rate of autologous stem cell transplantation (ASCT) for patients with MM is growing by approximately 3.3% annually.
Melphalan is the most commonly used IV agent for high-dose conditioning
for patients undergoing ASCT for MM. The current IV melphalan market is
EVOMELA was approved by
EVOMELA's melphalan formulation does not contain propylene glycol. The use of the Captisol® technology to reformulate also contributes to the 4-hour admixture stability of EVOMELA at room temperature. This is in addition to the 1 hour stability of reconstituted EVOMELA drug product at room temperature and 24 hour stability at refrigerated temperature (5°C).
Please see the Important Safety Information below and the full prescribing information, including BOXED WARNINGS, for EVOMELA at www.evomela.com.
Important Safety Information
WARNING: SEVERE BONE MARROW SUPPRESSION,
HYPERSENSITIVITY, and LEUKEMOGENICITY
- History of serious allergic reaction to melphalan.
Warnings and Precautions
- Nausea, vomiting, diarrhea or oral mucositis may occur. Provide supportive care using antiemetic and antidiarrheal medications as needed.
- Hepatic disorders ranging from abnormal liver function tests to clinical manifestations such as hepatitis and jaundice have been reported after treatment with melphalan. Hepatic veno-occlusive disease has also been reported. Monitor liver chemistries.
- EVOMELA can cause fetal harm when administered to a pregnant woman. Advise females of reproductive potential to avoid pregnancy during and after treatment with EVOMELA. If this drug is used during pregnancy or if the patient becomes pregnant while taking this drug, advise the patient of potential risk to the fetus.
- Melphalan-based chemotherapy regimens have been reported to cause suppression of ovarian function in premenopausal women, resulting in persistent amenorrhea in approximately 9% of patients. Reversible or irreversible testicular suppression has also been reported.
- The most common adverse reactions observed in at least 50% of patients with multiple myeloma treated with EVOMELA were neutrophil count decreased (100%), white blood cell count decreased (100%), lymphocyte count decreased (98%), platelet count decreased (98%), diarrhea (93%), nausea (90%), fatigue (77%), hypokalemia (74%), anemia (66%), and vomiting (64%).
- In a single-arm clinical study, twelve (20%) patients with multiple myeloma who received EVOMELA conditioning for ASCT experienced a treatment emergent serious adverse reaction. The most common serious adverse reactions ( > 1 patient, 1.6%) were pyrexia, hematochezia, febrile neutropenia, and renal failure.
- In a randomized clinical trial studying the palliative treatment of patients with multiple myeloma, severe myelotoxicity (WBC ≤1,000 and/or platelets ≤25,000) was more common in the IV melphalan arm (28%) than in the oral melphalan arm (11%).
- No formal drug interaction studies have been conducted. When nalidixic acid and IV melphalan are given simultaneously, the incidence of severe hemorrhagic necrotic enterocolitis has been reported to increase in pediatric patients.
Use in Specific Populations
- It is not known whether melphalan is present in human milk. Because many drugs are excreted in human milk and because of the potential for serious adverse reactions in nursing infants from melphalan, breastfeeding is not recommended during treatment with EVOMELA.
- Advise females of reproductive potential to avoid pregnancy, which may include the use of effective contraception methods, during and after treatment with EVOMELA.
- For Palliative Treatment, consider dose reduction for patients with renal impairment receiving EVOMELA.
Captisol is a patent-protected, chemically modified cyclodextrin with a
structure designed to optimize the solubility and stability of drugs.
Captisol was invented and initially developed by scientists in the
laboratories of Dr.
Spectrum Pharmaceuticals is a leading biotechnology company focused on acquiring, developing, and commercializing drug products, with a primary focus in Hematology and Oncology. Spectrum currently markets six hematology/oncology drugs, and expects an FDA decision on another drug in the second half of 2016. Additionally, Spectrum's pipeline includes two drugs targeting blockbuster markets in advanced stages of clinical development. Spectrum's strong track record for in-licensing and acquiring differentiated drugs, and expertise in clinical development have generated a robust, diversified, and growing pipeline of product candidates in advanced-stage Phase 2 and Phase 3 studies. More information on Spectrum is available at www.sppirx.com.
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