FDA Approval for Endogenous Cushing’s Syndrome Drug Recorlev

Posted on January 2, 2022 by MaryO

Ahead of its New Year’s Day decision deadline at the FDA, Xeris Biopharma has snagged an approval for Recorlev, a drug formerly known as levoketoconazole.

Based on results from phase 3 studies called SONICS and LOGICS, the FDA approved the drug for adults with Cushing’s syndrome. Xeris picked up Recorlev earlier this year in its acquisition of rare disease biotech Strongbridge Biopharma. It’s planning to launch in the first quarter of 2022.

Recorlev’s approval covers the treatment of endogenous hypercortisolemia in adults with Cushing’s syndrome who aren’t eligible for surgery or haven’t responded to surgery.

Endogenous Cushing’s disease is caused by a benign tumor in the pituitary gland that prompts the body to produce elevated levels of cortisol, which over time triggers a range of devastating physical and emotional symptoms for patients.

 

In the SONICS study, the drug significantly cut and normalized mean urinary free cortisol concentrations without a dose increase, according to the company. The LOGICS trial confirmed the drug’s efficacy and safety, Xeris says.

Cushion’s is a potentially fatal endocrine disease, and patients often experience years of symptoms before an accurate diagnosis, the company says. After a diagnosis, they’re presented with limited effective treatment options.

Following the approval, the company’s “experienced endocrinology-focused commercial organization can begin rapidly working to help address the needs of Cushing’s syndrome patients in the U.S. who are treated with prescription therapy,” Xeris CEO Paul R. Edick said in a statement.

Aside from its forthcoming Recorlev launch, Xeris markets Gvoke for severe hypoglycemia and Keveyis for primary periodic paralysis.

Back in October, the company partnered up with Merck to help reformulate some of the New Jersey pharma giant’s monoclonal antibody drugs.

From https://www.fiercepharma.com/pharma/xeris-biopharma-scores-fda-approval-for-endogenous-cushing-s-syndrome-drug-recorlev

Filed under: Cushing's, pituitary, Treatments | Tagged: , , , , | 1 Comment »

FDA Approves High-resolution MRI, Better at Spotting Pituitary Tumors in Cushing’s Patients

Posted on November 7, 2018 by MaryO

The U.S. Food and Drug Administration has approved the clinical use of a magnetic resonance imaging (MRI) scanner — the ultra-high-field 7T Terra MRI — with unprecedented resolution that allows for more reliable images of the brain.

The approach recently allowed the precise localization of a small tumor in the pituitary gland, which standard MRI had failed to spot, in a patient with Cushing’s disease.

So far, only one scanner of this kind exists in the U.S.. It was installed in February 2017 at the Mark and Mary Stevens Neuroimaging and Informatics Institute (INI) of the Keck School of Medicine, University of Southern California (USC).

The new scanner has an increased magnetic field strength of 7 Tesla, which is more than four times that of conventional MRI. This property greatly improves the instrument’s signal-to-noise ratio, dramatically increasing the spatial resolution and contrast of its images so that scientists can visualize the human living brain in high-definition and with unprecedented detail.

The 7T Terra is ideal for high-resolution neuroimaging, exploration of neurodegenerative diseases such as Alzheimer’s and Parkinson’s, and diagnosis and treatment of other brain diseases, a USC news story by Zara Greenbaum states.

Earlier this year, a report described the case of women with Cushing’s disease with a pituitary adenoma (slow-growing, benign tumor in the pituitary gland) that was possible to localize only with the new 7T MRI.

Based on laboratory analysis that revealed high levels of adrenocorticotropic hormone(ACTH) and cortisol, the doctors suspected a pituitary adenoma and recommended the patient for surgery. However, they ignored the precise location of the tumor, which failed to be detected by standard MRI scanners (1T and 3T).

    Two hours before surgery, the woman underwent a 7T MRI scan which finally identified with high precision the location of the adenoma, a very small tumor of 8 mm on the right side of the pituitary gland.

    “The 7T may save patients an invasive procedure. It also makes it easier for neurosurgeons to selectively remove a tumor without damaging surrounding areas,” said Gabriel Zada, MD, associate professor of neurological surgery at the Keck School.

    Since its arrival, the device has supported exploratory research into both healthy and diseased brains.

    Now the scanner’s advanced imaging technology can be used to help with diagnosis, treatment and monitoring of patients with neurological diseases, including Cushing’s disease.

    “This device, which has already made its mark as a powerful tool to advance research in the neurosciences, is now accessible to clinical populations in addition to researchers,” said Arthur W. Toga, PhD, provost professor and chair at the Keck School and director of the USC Stevens INI.

    “Clinicians across the university and beyond can now leverage all the benefits of increased spatial resolution to serve patients in need,” he said.

    Adapted from https://cushingsdiseasenews.com/2018/11/06/fda-oks-high-resolution-mri-better-spotting-pituitary-tumor-cushings/

    Filed under: Cushing's, Diagnostic Testing, pituitary | Tagged: , , , , | Leave a comment »

    [Pseudo-Cushing’s] Michigan woman nearly dies after herbal supplement found to be laced with steroids

    Posted on July 28, 2017 by MaryO

    MADISON HEIGHTS, Mich. (WXYZ) – Since 2004, the U.S. Food and Drug Administration has received more than 26,000 reports of adverse events and complaints about dietary supplements.

    Jody Higgins of Madison Heights, Michigan made one of those complaints to the FDA, after she says she found out the herbs she had been taking were making her seriously ill.

    “I really thought I was going to die I was getting so sick,” Higgins said.

    Back in 2015, Higgins says her legs started hurting.  She says she didn’t have great health insurance, and she was hoping for a more holistic approach, so a friend referred her to Far East Ginseng Herbs and Tea in nearby Sterling Heights.

    “They suggested that I take something that was called Linsen Double Caulis. I had never heard of it before, and it appeared to have all herbs on the label,” Higgins said.

    Higgins says for a while, she felt better, and when she stopped taking the Linsen Double Caulis, the leg pain returned. So, she says she kept taking it for nearly a year, even though she started noticing strange symptoms.

    “Within four months I had gained 80 pounds,” she said.

    She suddenly had facial hair growth, severe facial swelling, extremely swollen ankles, and had dark purple stretch marks all over her body.

    “I wasn’t recognizable,” said Higgins.  “I couldn’t stand for longer than 2 minutes. I couldn’t cook. I couldn’t wash my clothing. I could barely get in the shower.”

    After visiting several doctors, Higgins was eventually referred to University of Michigan Endocrinologist Dr. Ariel Barkan.

    “The minute that I said I had been taking a Chinese herbal remedy, he said ‘you’ve been poisoned. I know it.’ Those were his exact words,” said Higgins.

    “Her situation was pretty shaky,” Barkan said.

    Barkan sent the Linsin Double Caulis herbal supplement to the Mayo Clinic for testing.

    “They were loaded with Dexamethasone … [which] is a medication.  It’s a synthetic steroid, very potent, very long acting, and if we take it for quite some time, we develop what is called Cushing Syndrome,” said Dr. Barkan.

    Higgins was diagnosed with Cushing Syndrome, and Barkan says she could have died if she hadn’t sought help.

    “The mortality for untreated Cushing Syndrome is 50% within 5 years,” said Barkan.  “ … immunity is completely suppressed. And when you don’t have immunity, the first virus, the first germ may cause [a] fatal infection and you will die.”

    Higgins says once she stopped taking the Linsen Double Caulis, the facial hair went away, but she’s still struggling with her weight. Barkan says her health should improve, although it will take time.

    Both doctor and patient say they have contacted the FDA about this, and they each have a warning about taking herbal supplements.

    “Please just be very cautious,” Higgins said.

    “Don’t touch it. Don’t touch it, you’re playing Russian roulette,” said Barkan.

    Jody Higgins says she met with an investigator from the FDA’s criminal division.

    An FDA spokesperson would only say that they do not discuss possible or ongoing investigations.

    The lawyer for the store where Higgins says she purchased the supplement told us the owners will not be commenting on, but the owner did say they no longer sell this product.

    From http://www.fox4now.com/news/national/madison-heights-woman-herbal-supplement-caused-life-threatening-illness

    Filed under: Cushing's, symptoms | Tagged: , , , , , , , , , , , , , | Leave a comment »

    NDA for Macrilen™ for the Evaluation of Growth Hormone Deficiency in Adults

    Posted on July 19, 2017 by MaryO

    CHARLESTON, S.C.–(BUSINESS WIRE)–Aeterna Zentaris Inc. (NASDAQ: AEZS)(TSX: AEZS) (the “Company”) today announced that it has been notified by the U.S. Food and Drug Administration (“FDA”), that the Company’s New Drug Application (“NDA”) seeking approval of Macrilen™ (macimorelin) for the evaluation of growth hormone deficiency in adults (“AGHD”) has been accepted as a complete response to the FDA’s November 5, 2014 Complete Response Letter and granted a PDUFA date of December 30, 2017.

    David A. Dodd, President and Chief Executive Officer of the Company stated, “We are pleased that the FDA has formally accepted our resubmitted NDA and that it is under active review with an end-of-year PDUFA date. We remain confident that the FDA will approve our NDA and, therefore, we are moving forward with our preparations to launch the product in the first quarter of 2018.”

    The Company also announces that Mr. Kenneth Newport is no longer a member of the Board of Directors effective as of July 12, 2017.

    About MacrilenTM (macimorelin)

    Macimorelin, a ghrelin agonist, is an orally-active small molecule that stimulates the secretion of growth hormone. Macimorelin has been granted orphan drug designation by the FDA for diagnosis of AGHD. The Company owns the worldwide rights to this patented compound and has significant patent protection left. The Company’s U.S. composition of matter patent expires in 2022 and its U.S. utility patent runs through 2027. The Company proposes, subject to FDA approval, to market macimorelin under the tradename Macrilen™.

    About AGHD

    AGHD affects approximately 75,000 adults across the U.S., Canada and Europe. Growth hormone not only plays an important role in growth from childhood to adulthood, but also helps promote a hormonally-balanced health status. AGHD mostly results from damage to the pituitary gland. It is usually characterized by a reduction in bone mineral density, lean body mass, exercise capacity, and overall quality of life as well as an increase of cardiovascular risks.

    About Aeterna Zentaris Inc.

    Aeterna Zentaris is a specialty biopharmaceutical company engaged in developing and commercializing novel pharmaceutical therapies. We are engaged in drug development activities and in the promotion of products for others. We recently completed Phase 3 studies of two internally developed compounds. The focus of our business development efforts is the acquisition of licenses to products that are relevant to our therapeutic areas of focus. We also intend to license out certain commercial rights of internally developed products to licensees in non-U.S. territories where such out-licensing would enable us to ensure development, registration and launch of our product candidates. Our goal is to become a growth-oriented specialty biopharmaceutical company by pursuing successful development and commercialization of our product portfolio, achieving successful commercial presence and growth, while consistently delivering value to our shareholders, employees and the medical providers and patients who will benefit from our products. For more information, visit www.aezsinc.com.

    Forward-Looking Statements

    This press release contains forward-looking statements made pursuant to the safe harbor provision of the U.S. Securities Litigation Reform Act of 1995, which reflect our current expectations regarding future events. Forward-looking statements may include, but are not limited to statements preceded by, followed by, or that include the words “expects,” “believes,” “intends,” “anticipates,” and similar terms that relate to future events, performance, or our results. Forward-looking statements involve known risks and uncertainties, many of which are discussed under the caption “Key Information – Risk Factors” in our most recent Annual Report on Form 20-F filed with the relevant Canadian securities regulatory authorities in lieu of an annual information form and with the U.S. Securities and Exchange Commission (“SEC”). Such statements include, but are not limited to, statements about the progress of our research, development and clinical trials and the timing of, and prospects for, regulatory approval and commercialization of our product candidates, the timing of expected results of our studies, anticipated results of these studies, statements about the status of our efforts to establish a commercial operation and to obtain the right to promote or sell products that we did not develop and estimates regarding our capital requirements and our needs for, and our ability to obtain, additional financing. Known and unknown risks and uncertainties could cause our actual results to differ materially from those in forward-looking statements. Such risks and uncertainties include, among others, the availability of funds and resources to pursue our research and development projects and clinical trials, the successful and timely completion of clinical studies, the risk that safety and efficacy data from any of our Phase 3 trials may not coincide with the data analyses from previously reported Phase 1 and/or Phase 2 clinical trials, the rejection or non-acceptance of any new drug application by one or more regulatory authorities and, more generally, uncertainties related to the regulatory process (including whether or not the regulatory authorities will definitively accept the Company’s conclusions regarding Macrilen™ and approve its registration following the Company’s re-submission of an NDA for the product as described elsewhere in this press release), the ability of the Company to efficiently commercialize one or more of its products or product candidates, the degree of market acceptance once our products are approved for commercialization, our ability to take advantage of business opportunities in the pharmaceutical industry, our ability to protect our intellectual property, and the potential of liability arising from shareholder lawsuits and general changes in economic conditions. Investors should consult the Company’s quarterly and annual filings with the Canadian securities commissions and the SEC for additional information on risks and uncertainties. Given these uncertainties and risk factors, readers are cautioned not to place undue reliance on these forward-looking statements. We disclaim any obligation to update any such factors or to publicly announce any revisions to any of the forward-looking statements contained herein to reflect future results, events or developments, unless required to do so by a governmental authority or applicable law.

    Contacts

    Aeterna Zentaris Inc.
    Philip A. Theodore, 843-900-3211
    Senior Vice President
    ir@aezsinc.com

    From http://www.businesswire.com/news/home/20170718006321/en/NDA-Macrilen%E2%84%A2-Evaluation-Growth-Hormone-Deficiency-Adults

    Filed under: Diagnostic Testing, growth hormone | Tagged: , , , , | Leave a comment »

    RDLA’s May Legislative In-Person Meeting

    Posted on May 4, 2016 by MaryO

    RDLA Congressional Caucus

     

    Rare Disease Legislative Advocates in coordination with Rare Disease Congressional Caucus Co-Chairs: Representative Leonard Lance (R-NJ), Representative Joe Crowley (D-NY), Senator Orrin Hatch (R-UT), and Senator Amy Klobuchar (D-MN); and the Office of Senator Mark Kirk (R-IL) will host a briefing on:

     The NIH and FDA: Vital Agencies in the Fight Against Rare Diseases

    Wednesday, May 18th, 2016

    2:00 pm – 3:00 pm

    Senate Capitol Visitors Center Room 201, Washington, D.C., 20004

       REGISTER   

    Moderator:  Ellie Dehoney, Vice President of Policy and Advocacy, Research!America

    • The Undiagnosed Disease Program at the NIH
      • William Gahl, M.D., Ph.D, Clinical Director, National Human Genome Research Institute (invited)
    • Precision Medicine – The White House & the NIH
      • Matthew Might, Strategist, Executive Office of the President, The White House, Associate Professor, University of Utah, Associate Professor, Visiting, Harvard Medical School, Founder, NGLY1.org (invited)
    • The Value of Patients to Clinical Innovation at the NIH
      • Kayla Martinez & Dorelia Rivera, NOMID Patient
    • The Role of NIH Funding in Kickstarting Biomedical Innovation
      • Christopher C. Gibson, Ph.D, Co-Founder & CEO, Recursion Pharmaceuticals

    Come and enjoy an array of refreshments  or have coffee and snack break while learning about the role of our health agencies.

    Filed under: Meetings and Conferences, Rare Diseases | Tagged: , , , , , , , | Leave a comment »

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