First FDA Approval for Sickle Cell Treatment in Nearly 20 Years

The U.S. Food and Drug Administration approved Endari (L-glutamine oral powder) for patients age five years and older with sickle cell disease to reduce severe complications associated with the blood disorder.? The FDA granted the approval of Endari to Emmaus Medical Inc.

“Endari is the first treatment approved for patients with sickle cell disease in almost 20 years,” said Richard Pazdur, M.D., acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research and director of the FDA’s Oncology Center of Excellence. “Until now, only one other drug was approved for patients living with this serious, debilitating condition.”

Sickle cell disease is an inherited blood disorder in which the red blood cells are abnormally shaped (in a crescent, or “sickle,” shape). This restricts the flow in blood vessels and limits oxygen delivery to the body’s tissues, leading to severe pain and organ damage. According to the National Institutes of Health, approximately 100,000 people in the United States have sickle cell disease. The disease occurs most often in African-Americans, Latinos and other minority groups. The average life expectancy for patients with sickle cell disease in the United States is approximately 40 to 60 years.

The safety and efficacy of Endari were studied in a randomized trial of patients ages five to 58 years old with sickle cell disease who had two or more painful crises within the 12 months prior to enrollment in the trial. Patients were assigned randomly to treatment with Endari or placebo, and the effect of treatment was evaluated over 48 weeks. Patients who were treated with Endari experienced fewer hospital visits for pain treated with a parenterally administered narcotic or ketorolac (sickle cell crises), on average, compared to patients who received a placebo (median 3 vs. median 4), fewer hospitalizations for sickle cell pain (median 2 vs. median 3), and fewer days in the hospital (median 6.5 days vs. median 11 days).? Patients who received Endari also had fewer occurrences of acute chest syndrome (a life-threatening complication of sickle cell disease) compared with patients who received a placebo (8.6 percent vs. 23.1 percent).

Endari received Orphan Drug designation for this use, which provides incentives to assist and encourage the development of drugs for rare diseases.? In addition, development of this drug was in part supported by the FDA Orphan Products Grants Program, which provides grants for clinical studies on safety and/or effectiveness of products for use in rare diseases or conditions.

Displayed with permission from PRNewswire for Journalists; Illustration Courtesy of NIH

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Clinical Trials Monthly Case Study Analysis: Cost Reductions / Hematology

Each month, we investigate a different Clinical Trial Case Study that posed us with a challenging question; this time it’s a look at an orphan indication in HEMATOLOGY, and significant cost reductions for the sponsor:

We asked ourselves:

Scenario: A Phase IV study was required by the FDA as a condition of approval of an orphan drug. The sponsor was faced with monitoring, managing, and locking data for a 12-month study conducted at 100 sites.

Criterium offered an integrated technology-enhanced solution that allowed the sites to register ePRO Patient Diaries Input-Output Diagrampatients and allowed the patients to record their event data directly into our remote systems.

Criterium’s solution enabled the client to eliminate most of the scheduled monitoring visits. Criterium’s diligence and centralized system meant that monitors only had to visit sites that had problems.

What If: If the client had used traditional methods, the cost would have soared for a product that has modest revenue and it would have taken much longer to provide the data to the FDA and the prescribing community.

Results: Criterium’s centralized real-time data accession utilizing Automated CRFs and Interactive Voice Response (IVR) medical history and patient diary application saved the client $1 million to $1.5 million in clinical monitoring and data management costs. In addition, the data were satisfactory for rapid submission to the FDA for conditional approval, were provided to the medical community to support the indication for which the drug was approved and numerous publications resulted from this fairly large body of data for this orphan indication.

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Halozyme: Update Following Type B FDA Meeting

Recent FDA meeting allows for potential marketing application based on progression free survival in pivotal Phase 3 pancreatic cancer study

Halozyme Therapeutics, Inc.,a biotechnology company developing novel oncology and drug-delivery therapies, today announced it plans to proceed with a Phase 3 clinical study (Study 301) of its investigational new drug PEGPH20 in patients with metastatic pancreatic cancer, using a design allowing for potential marketing application based on either progression free survival (PFS) or overall survival. The use of PFS as the basis for marketing approval will be subject to the overall benefit and risk associated with PEGPH20 combined with nab-paclitaxel (ABRAXANE ?) and gemcitabine therapy, including the:

  • Magnitude of the PFS treatment effect observed;
  • Toxicity profile;?and
  • Interim overall survival data.

The study will enroll patients whose tumors accumulate high levels of?hyaluronan?(HA)?? a?sugar?that?is sometimes more prevalent in the areas surrounding cancer cells. Halozyme recently discussed the study as part of a planned Type B meeting with the U.S. Food and Drug Administration (FDA).

bullseyeData discussed at the meeting focused on interim results from the company’s randomized Phase 2 study in pancreatic cancer, Study 202, which showed a doubling in median PFS in metastatic pancreatic cancer patients with high levels of HA who were treated with Halozyme’s investigational new drug PEGPH20 combined with nab-paclitaxel (ABRAXANE ?) and gemcitabine (9.2 months vs. 4.3 months in patients treated with nab-paclitaxel ABRAXANE ? and gemcitabine alone). The potential risk profile, including rate of thromboembolic events, was also discussed.? Additional?takeaways from the meeting include:

  • Halozyme?affirmed?plans?to enroll and evaluate high-HA patients using a companion diagnostic test; and
  • FDA provided feedback on the current?companion diagnostic approach and confirmed that an approved companion diagnostic strategy is required prior to Phase 3 study initiation.

“We are encouraged by the FDA’s feedback and their willingness to consider a novel primary endpoint such as PFS given the high unmet medical need in high-HA metastatic pancreatic cancer,” said Dr. Helen Torley, President and Chief Executive Officer. “We are committed to rapidly finalizing the Phase 3 protocol and developing the companion diagnostic regulatory filings required to initiate Study 301.” Based on this FDA feedback, the company is targeting the end of first quarter 2016 to initiate the Phase 3 study.

About Study 202 & PEGPH20microscope in laboratory
Study 202 (Halo 109-202) is a Phase 2 multi-center, randomized clinical trial evaluating investigational new drug PEGPH20 as a first-line therapy for treatment of patients with stage 4 metastatic pancreatic cancer. The primary outcome of the trial is to measure improvement in progression-free survival in patients receiving investigational new drug PEGPH20 in combination with gemcitabine and nab-paclitaxel compared to gemcitabine and nab-paclitaxel alone. A second primary endpoint will assess the thromboembolic event rate in the PEGPH20 treatment arm. Secondary endpoints also include objective response rate and overall survival. More information may be found at:

PEGPH20 is an investigational PEGylated form of Halozyme’s proprietary recombinant human hyaluronidase under clinical development for the systemic treatment of tumors that accumulate hyaluronan. FDA granted orphan drug designation to PEGPH20 for treatment of pancreatic cancer and fast track for PEGPH20 in combination with gemcitabine and nab-paclitaxel for the treatment of metastatic pancreatic cancer. Additionally, the European Commission, acting on the recommendation from the Committee for Orphan Medicinal Products of the European Medicines Agency, designated investigational drug PEGPH20 an orphan medicinal product for the treatment of pancreatic cancer.

About Halozyme
Halozyme Therapeutics is a biotechnology company focused on developing and commercializing novel oncology therapies that target the tumor microenvironment. Halozyme’s lead proprietary program, investigational drug PEGPH20, applies a unique approach to targeting solid tumors, allowing increased access of co-administered cancer drug therapies to the tumor.? PEGPH20 is currently in development for metastatic pancreatic cancer and non-small cell lung cancer and has potential across additional cancers in combination with different types of cancer therapies. For more information, visit

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FDA Approves Pulmozyme with eRapid Nebulizer

CF Patients See 2 – 3 Minute Treatment Times

The eRapid Nebulizer System (eRapid) from PARI has been approved as the first electronic nebulizer by the Food and Drug Administration to deliver Genentech’s Pulmozyme for cystic fibrosis treatment.? A huge improvement for cystic fibrosis patients, eRapid is able to reduce average treatment times with Pulmozyme from 6-8 minutes down to 2-3 minutes.? Both adult and pediatric patients showed a strong preference for eRapid over conventional nebulizers in a clinical study. FDA-Logo

“We have been pleased with eRapid’s fast treatment times in the lab and are excited that patients now have access to a much faster Pulmozyme therapy.? As the first electronic nebulizer to deliver Pulmozyme, eRapid is a true breakthrough for cystic fibrosis patients who take the therapy daily, often for years,”?said Lisa Cambridge, director of Medical Science and Pharmaceutical Alliances at PARI Respiratory Equipment, Inc.

“PARI was motivated to introduce eRapid to the US market based on encouragement from the Cystic Fibrosis Foundation and their input to have a general-use, electronic nebulizer that could improve therapy adherence. For many years, eRapid has been successfully distributed in Europe (as eFlow Rapid) with favorable feedback from patients with CF. In the Pulmozyme clinical trial, there was a 10:1 preference for eRapid in the pediatric group and a 20:1 preference in the adult group.? That confirmed our decision to bring eRapid to the US,” added Geoff A. Hunziker, president of PARI USA.

“After the successful results of a Phase IV study, we are confident that physicians will see that both pediatric and adult patients favor eRapid based on reduced treatment times, quiet operation, and its small, portable size.? We were also happy to see that patients were more satisfied with treatment and eRapid had a positive influence on adherence ? good for their overall cystic fibrosis management,” added Lisa Cambridge.? eRapid is available today by prescription through a select group of specialty pharmacies.? Visit or call 1-800-FAST-NEB to learn more.

Pulmozyme is indicated for daily administration along with standard therapies for the management of cystic fibrosis to improve pulmonary function.? For more information, visit

About eRapid Nebulizer System eRapid is an electronic nebulizer that enables quick and efficient inhalation that can greatly reduce a patient’s treatment burden. ?For more information on eRapid, please visit

About Cystic Fibrosis Cystic fibrosis, a life-threatening disease affecting 30,000 American patients, involves a genetic mutation that results in poorly hydrated, thickened mucus secretions in the lungs, as well as severely impaired mucociliary clearance.? Get more information about Cystic Fibrosis, visit

About PARI Respiratory Equipment, Inc. PARI is a leading, worldwide developer and manufacturer of fast and efficient aerosol delivery systems for patients with asthma, chronic lung disease, cystic fibrosis, RSV, VAP, and HAP.? PARI’s worldwide vision is to improve the lives of those affected by respiratory diseases and those who care for them.?PARI is considered the gold standard for aerosol delivery for nebulizer therapies.? Featured products include the PARI LC PLUS Reusable Nebulizer, Vortex Holding Chamber, and the drug-specific eFlow Technology platform.

SOURCE: PARI Respiratory Equipment, Inc., Displayed with permission from PR Newswire via RePubHub

Multiple Myeloma Compound Gets Orphan Drug Status

Dr J S Macdonald MDDr. John S. Macdonald
Senior Consultant for The Academic GI Cancer Consultant Consortium (AGICC) & The Academic Myeloma Consortium (AMyC) of The Oncology Consortia of CRITERIUM, INC.

The US Food and Drug Administration has granted orphan drug status to an experimental compound for multiple myeloma known as KRN5500.

This is the second orphan drug designation for KRN5500, which is a novel, intravenous, nonopioid, nonnarcotic compound in phase 2 clinical development by DARA BioSciences.

Earlier this year, KRN5500 received orphan status for the treatment of chemotherapy-induced neuropathic pain refractory to conventional analgesics in patients with cancer.

“It is noteworthy in this regard that up to 20% of myeloma patients have intrinsic peripheral neuropathy, an incidence that increases to the range of 75% in patients treated with neurotoxic drugs such as thalidomide or bortezomib,” said David J. Drutz, MD, chief executive officer and chief medical officer of DARA BioSciences, in a statement.

In 2011, KRN5500 was granted a fast-track designation, a process designed to facilitate development and to expedite the review of drugs to treat serious conditions and fill an unmet medical need.FDA-Logo

The therapeutic potential of KRN5500 has been demonstrated in vitro and in vivo. The agent, which is a spicamycin derivative, exhibits antimyeloma effects by impairing both myeloma cells and osteoclasts, the company reports.

The orphan drug designation is “an important step toward the potential treatment of multiple myeloma and one of its major complications,” Dr. Drutz said. It will also help in the company’s “ongoing pursuit of partnering opportunities to assist in funding the clinical advancement and development pathway of KRN5500.”

Dr. John S. Macdonald comments on this important FDA decision…
“The fast tracking of this spicamycin derivative recognizes an agent that not only possesses anti cancer (multiple myeloma) activity likely to be beneficial in patients resistant to standard treatment, but also has the potential to decrease a very serious complication of myeloma, bone fractures because it inhibits osteoclast activity.”

SOURCE: Original article by Megan Brooks for MedScape –

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