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In the  current fortnightly edition of Medness Plus, Esha Sehanobish reports on FDA’s approval Arikayce, the first and only therapy specifically indicated for the treatment of Mycobacterium Avium Complex (MAC) Lung Disease in adult patients with limited treatment options. Also included are FDA approvals of an over-the-counter hearing aid from Bose, Progenika Biopharma’s DNA-Based assay kit that could make Blood Transfusions safer, Roche’s blockbuster drug Hemlibra which reduces bleeds and administration frequencies in hemophilia A patients. Another approval that is potentially helping reduce hospitalization costs is Paratek’s antibiotic Nuzyra, which offers clinicians an option for IV treatment of community-acquired bacterial pneumonia (CABP) and acute skin and skin structure infections (ABSSI) patients as well as transition them home to complete treatment with the oral formulation.- Abhi Dey

  1. FDA approves an antibacterial drug, Arikayceto treat severe lung disease.

FDA recently approved Arikayce, a new drug, for the treatment of severe lung disease caused by a group of Mycobacterium avium complex (MAC) usually in a limited number of patients who are unresponsive to the conventional treatment (refractory disease) [1]. Arikayce is an amikacin liposome inhalation suspension) and is a product of Insmed, Inc. of Bridgewater, NJ.

MAC is usually found in soil and water and is a nontuberculous variety of Mycobacterium. Some of the common symptoms of the lung disease caused by this group of bacteria include fatigue, persistent cough, night sweats and occasional shortness of breath and coughing up of blood. Arikayce won an approval under FDA’s Accelerated Approval pathway. Under this pathway, FDA usually approves the drugs for life-threatening diseases if it has shown significant effects on surrogate endpoints and if there are sufficient evidence of the drug being clinically beneficial to patients in the future. The decision was an outcome of the negative results obtained from monthly sputum cultures of patients by month six of the treatment. It is an inhaled treatment taken in through a nebulizer. The study was a part of a randomized, clinical trial where patients were subjected to one of the two available treatments. For one group, along with Arikayce, there was a multi-drug antibacterial regimen and for the other group, there was only the latter. Six months into the treatment 29 percent of the patients treated with Arikayce showed no growth of Mycobacterium in their sputum cultures for the next three months compared to just 9 percent for those not treated with the drug. A boxed warning has also been issued with the prescribing information of Arikayce which includes an increased risk of respiratory conditions such as bronchospasm, hypersensitivity pneumonitis, hemoptysis and exacerbation of underlying lung disease. Other common side-effects are dysphonia, ototoxicity, musculoskeletal pain, diarrhea, upper airway irritation, fatigue and nausea.


Further post-market studies describing the clinical benefits of Arikayce needs to be conducted by the sponsors of the drug as a requirement by the FDA. Arikayce is also the first drug to be approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs or LPAD pathway. Established by Congress under the 21st Century Cures Act to allow and advance the development of antifungal and antibacterial drugs for the treatment of serious or life-threatening infections in patients unresponsive to the available ones. These programs usually involve shorter or fewer clinical trials and the drugs approved under these pathways includes statements that convey that the drug has been shown to be effective and safe only for a limited population.


“As bacteria continue to grow impervious to currently available antibiotics, we need to encourage the development of drugs that can treat resistant infections. That means utilizing novel tools intended to streamline development and encourage investment into these important endeavors,” said FDA Commissioner Scott Gottlieb, M.D. “This approval is the first time a drug is being approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs, and it marks an important policy milestone. This pathway, advanced by Congress, aims to spur development of drugs targeting infections that lack effective therapies. We’re seeing a lot of early interest among sponsors in using this new pathway, and it’s our hope that it’ll spur more development and approval of antibacterial drugs for treating serious or life-threatening infections in limited populations of patients with unmet medical needs.”

 

  1. FDA approves the expansion of the use of Gardasil 9 to include patients between the age of 27 through 45 years.

 


FDA recently approved a supplemental application for Gardasil 9, a recombinant Human Papillomavirus (HPV) 9-valent vaccine. Based on the application, the vaccine can now be extended to women and men aged 27 through 45 years. Gardasil 9, is known to prevent certain diseases caused by the nine HPV types covered by the vaccine [2]. The approval was granted to the Gardasil 9 Biologics License Application to Merck, Sharp and Dohme Corp. a subsidiary of Merck and Co., Inc.

HPV is associated with several forms of cancer and is gender independent. Data from CDC shows that every year about 14 million Americans become infected with HPV out of which 12000 women are diagnosed with cervical cancer and about 4000 of them die due to the same caused by HPV. The number is alarming, hence a vaccine with an effective age and gender range is desirable. Gardasil was approved in 2006 and was used to prevent certain diseases and cancers caused by four HPV types. It is no longer distributed in the US. Gardasil 9 was approved in 2014 and was known to cover an additional five types of HPV. It was approved for males and females between the age of 9 and 26. FDA’s approval of Gardasil 9 in women between the age of 27 and 45 was a result of the new data from a long-term follow-up study. The study involved approximately 3200 women 27 through 45 years of age, followed by an average of 3.5 years. Gardasil was found to be 88 percent effective in the prevention of combined endpoints consisting of genital warts, persistent infection, vaginal and vulvar precancerous lesions, cervical precancerous lesions and cervical cancer related to HPV infections covered by the vaccine. In men aged 27 through 45, the effectiveness of Gardasil 9 was obtained from the data for the women population and younger men, 16 through 26 years of age and from immunogenicity data from a clinical trial involving 150 men of the same age group receiving a 3-dose regimen of the vaccine over six months. The safety of the vaccine was evaluated for 13000 males and females. The most common side effects include swelling, redness, injection and injection site pain. The application for Gardasil 9 was given a priority review status under which the review of medical products with potential treatment applications to life-threatening diseases is expedited.

Proof Everyone Should Get The HPV Vaccine

 

“Today’s approval represents an important opportunity to help prevent HPV-related diseases and cancers in a broader age range,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. “The Centers for Disease Control and Prevention has stated that HPV vaccination prior to becoming infected with the HPV types covered by the vaccine has the potential to prevent more than 90 percent of these cancers, or 31,200 cases every year, from ever developing.”

 

 

  1. FDA announces the approval of NUZYRA (Omadacycline) a product of Paratek.


Paratek Pharmaceuticals recently announced that their product, NUZYRA received FDA approval for the treatment of community-acquired bacterial pneumonia (CABP) and acute skin and skin structure infections (ABSSI) [3]. It is a once-daily IV and oral antibiotic that is effective against a wide range of gram-positive, negative and drug-resistant strains of bacteria. The tetracycline is scheduled to reach the market in the first quarter of 2019.

NUZYRA (omadacycline) is a unique antibiotic which can be used both as an oral formulation and as a once-daily intravenous (IV) form for the treatment of ABSSSI and CABP. It is a form of tetracycline that was designed to overcome tetracycline resistance and be effective against a vast spectrum of bacteria.

It is used for the treatment of adults with infections caused by susceptible microorganisms such as Streptococcus pneumonia, Staphylococcus aureus, Haemophilus influenzae causing CABP and those that cause ABSSSI such as Klebsiella pneumoniae, Streptococcus pyogenes etc. The data from the Center for Disease Control and Prevention (CDC) estimates that 2 million illnesses caused by drug-resistant bacteria lead to approximately 23,000 deaths every year in the United States. Streptococcus pneumoniae causing CABP, results in 1.2 million infections and 7000 deaths whereas ABSSSI results in 750,000 hospitalizations. The approval comes from multiple clinical trials within Paratek’s global development program. Around 2,000 adults received NUZYRA and it was found to be generally safe and well-tolerated. Paratek has agreed to carry out post-marketing analysis in CABP and pediatrics. The most common adverse reactions are vomiting, nausea, hypertension, insomnia, diarrhea, constipation, headache, infusion-site reactions, alanine aminotransferase increase and increased gamma-glutamyltransferase.

Why is the Pneumonia still a serious risk to Hospital Patients

From Visually.

 

“The approval of NUZYRA is an historic milestone for Paratek as it represents 20 years of research and development of this life-saving antibiotic for patients affected by community-acquired infections,” said Michael F. Bigham, Chairman and CEO, Paratek. “There are countless champions of NUZYRA who have been tireless in their efforts to ensure its advancement to commercialization – from patients, clinicians and study investigators to our Paratek team. We are grateful to all who played a role in making NUZYRA available to patients in need. We are excited to launch NUZYRA early next year.” “In the face of ever-increasing antibiotic resistance, the FDA approved NUZYRA with a label having full approval for both CABP and ABSSSI.  We are excited to bring to physicians an effective, well-tolerated monotherapy option for patients,” said Evan Loh, M.D., President, Chief Operating Officer, and Chief Medical Officer, Paratek. “NUZYRA offers clinicians the ability to treat patients with the IV and transition them home to complete treatment with the oral formulation. This potentially helps reduce hospitalizations and the costs associated with hospital stays.”

 

  1. FDA approves Hemlibra for hemophilia A without factor VIII inhibitors, a product of Roche.


FDA recently announced the approval of Roche’s Hemlibra (emicizumab-kxwh) for prophylaxis to prevent or reduce the frequency of bleeding episodes in children and adults and newborns and older people with hemophilia A without VIII factor inhibitors [4]. For those with hemophilia A with and without VIII factor inhibitor, Hemlibra is the only available prophylactic treatment available. It can be administered subcutaneously and in multiple doses (once weekly, every two or four weeks). The approval was based on the positive results from the phase III, HAVEN 3 and HAVEN 4 studies. Hemophilia prophylaxis showed statistically significant and clinically meaningful reductions in treated bleeds as compared to no prophylaxis (primary endpoints).

In November 2017, FDA approved Hemlibra for children and adults with hemophilia A with the factor VIII inhibitors. Since then it has also been studied as a part of the largest pivotal clinical trial programs in people with hemophilia A with and without factor VIII inhibitors. Hemlibra is a IXa and X factor-directed antibody. It coordinates the bringing together of the factor IXa and X proteins required to activate the natural coagulation system and restore the blood clotting process for patients with Hemophilia A. Hemophilia A is a serious inherited disorder in which the person’s blood does not clot properly followed by uncontrolled bleeding. Approximately 50% of the people with this disorder have a severe manifestation of the same. These people generally lack or have insufficient clotting protein factor VIII due to which it cannot result in bringing together the IXa and X factors which are involved in blood clotting. Based on the severity of the condition, hemophilia A patient’s can frequently bleed into their joints and muscles. Hemlibra was originally created by Chugai Pharmaceuticals and is now being co-developed by Chugai, Roche and Genentech. Genentech in the United States markets it. It is a preventive treatment that can be administered subcutaneously once a week, or every two or four weeks. The approval was based on the data from two clinical trials HAVEN 3 and HAVEN 4. HAVEN 3 is a randomized, open-label, multicenter, phase III study evaluating the safety, efficacy and pharmacokinetics of Hemlibra prophylaxis vs. the one without it, in patients with hemophilia A without factor VIII inhibitors. This study involved 152 patients above the age of 12 years. HAVEN 4 is a single-arm, multicenter, open-label, phase III study for evaluating the safety, pharmacokinetics and efficacy of the subcutaneous administration of Hemlibra every four weeks. 48 patients with hemophilia with or without VIII factor and above the age of 12 years were included in the study. In both the clinical trials, the primary endpoints were satisfactorily reached.

“Today’s approval of Hemlibra reflects our commitment to groundbreaking science and the development of medicines with the potential to redefine the standard of care,” said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. “Hemlibra is now the only FDA-approved medicine for people with haemophilia A with and without factor VIII inhibitors, based on the efficacy and safety profile demonstrated across four pivotal studies. We want to thank the haemophilia community for their partnership in helping us bring this new option to everyone living with haemophilia A.” “Many preventative treatment options for people with haemophilia A without factor VIII inhibitors require intravenous infusions several times a week. Even then, people can still experience bleeds, and there has been a need for more treatment options,” said Michael Callaghan, MD, haematologist, Children’s Hospital of Michigan, Detroit. “The approval of Hemlibra is an important advancement for the entire haemophilia A community, as we now have a new class of medicine for the first time in nearly 20 years. Hemlibra can reduce bleeds, and it offers a new subcutaneous administration once weekly, every two weeks or every four weeks.”

 

  1. FDA approves the marketing of the first self-fitting hearing aid, controlled by the user.


FDA recently announced the approval for marketing a new device, the Bose Hearing Aid, which intends to amplify the sounds for individuals 18 years or above with mild to moderate hearing loss [5]. This will let users fit, control and correctly program the hearing aid on their own without support from a healthcare provider.  The marketing rights of the Bose Hearing Aid device was given to Bose Corporation.

The device was reviewed under the FDA’s De Novo premarket review pathway. This pathway includes some low-to moderate-risk devices that are unique and novel and do not have any prior legally available and marketed device. Data shows that approximately 37.5 million adults over the age of 18 years have trouble in hearing and the degree of uneasiness varies widely. Various reasons can contribute to the hearing impairment including age, medical conditions and exposure to loud noises. Hearing-aids help such individuals hear speech and sound and communicate with the others. Thus, a user-fitted wireless air conduction hearing aid has been created by Bose. The concept of air conduction hearing uses sound vibrations through one or more microphones. The obtained signal is processed, amplified and played back through earphones in the ear canal. Using a mobile application in their phones, the patients can adjust the hearing aid. The adjustments are made by the user based on real-time and real-world experience, without the assistance of a healthcare professional. Even though the device is user-regulated, the device must comply with the federal and state laws regarding the hearing-aid. An example of such a state law is regarding the purchase of hearing aids from licensed hearing-aid dispenser. As required by the FDA reauthorization Act of 2017, they are still in the process of drafting proposed regulations for a new category of over-the-counter hearing aids. The approval was given based on clinical data from 125 patients, where self-fittings of the Bose hearing-air was comparable on average to those with professional fitting of the same device in the context of the speech in noise testing, amplification selected and overall benefit. It was observed that participants preferred self-fitted settings over the professional-fitted ones. The Bose Hearing Aid has also been labeled to inform patients when to consult a hearing health professional.

Hearing Loss in America

From Visually.

 

“Hearing loss is a significant public health issue, especially as individuals age,” said Malvina Eydelman, M.D., director of the Division of Ophthalmic, and Ear, Nose and Throat Devices at the FDA’s Center for Devices and Radiological Health. “Today’s marketing authorization provides certain patients with access to a new hearing aid that provides them with direct control over the fit and functionality of the device. The FDA is committed to ensuring that individuals with hearing loss have options for taking an active role in their health care.”

 

  1. To determine blood compatibility, FDA approves a new DNA-based test.


FDA recently approved ID CORE XT, a molecular-based assay used in blood transfusion medicine to determine blood compatibility. The technique aims to assess blood donor and patient non-ABO red blood cell types [6]. It is the first to report genotypes as final results and the second molecular assay approved in transfusion medicine. The approval for the ID CORE XT test was granted to Progenika Biopharma S.A., a Grifols company.

Based on the antigens present on the surface of the red blood cells, human blood can be classified into different groups. Along with the ABO blood group antigens, the presence or absence of other blood group antigens is important when matching blood during transfusions since some people develop antibodies to non-ABO antigens. People who receive frequent blood transfusions such as those with sickle cell anemia are more prone to develop such antibodies. If there is an incorrect match between red blood cells and non-ABO antigens during transfusion, an adverse transfusion reaction and red blood cell destruction are likely to occur. Red blood cell antigens are usually identified using serological methods with antisera, a blood serum that contains antibodies for testing. This type of testing can be scarce and unavailable hence has its limitations. The study that was conducted compared the typing results of the ID CORE XT Test with licensed serological reagents, the first FDA-approved molecular assay and DNA sequencing tests. The data showed similar performance between the methods.

“The approval of the ID CORE XT Test can streamline blood compatibility testing and provides an additional alternative to testing blood with antisera,” said Peter Marks, M.D., Ph.D., M.D., director of the FDA’s Center for Biologics Evaluation and Research. “We know that DNA testing holds great promise – to provide more informative, accurate and cost-effective methods that can enhance patient care.”

Blood. Simple.

From Visually.

 

 

Reference:

 

[1] https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm622048.htm.

[2] https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm622715.htm.

[3]http://investor.paratekpharm.com/phoenix.zhtml?c=253770&p=irolnewsArticle&cat=news&id=2369985/.

[4] https://www.roche.com/media/releases/med-cor-2018-10-04c.htm.

[5] https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm622692.htm.

[6] https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm623195.htm.

 

 

About the Author:

Esha Sehanobish

She is presently a Postdoctoral research fellow at Albert Einstein college of medicine, NY and works on characterization of enzymes that could act as potential therapeutic targets against tuberculosis. She is an enzymologist with a doctoral degree from the University of Central Florida in 2016. She loves using her communication skills to raise awareness about the importance of science in general by using social media. When she is not doing “science”, she loves designing and painting as a way of expressing ones thoughts through graphics and color.

 

Editor and Blog Design

Abhi Dey

Abhi graduated from the Molecular Biophysics Unit of IISc (Bangalore, India) in 2011. As a Biomedical Scientist, he has worked with all three life-forms in his 13-year research career, viz., particulate, unicellular and multicellular. He is currently an Assistant Scientist at Emory University (Atlanta, GA) studying mechanisms of tumor recurrence in kids with brain tumors. As a postdoctoral fellow, he was the recipient of two Young Investigator Awards from Alex Lemonade Stand Foundation (Philadelphia, PA) and Rockland Immunochemicals. His current research has been funded by Northwestern Mutual Foundation (Milwaukee, WI), CURE Childhood Cancer Foundation (Atlanta, GA) and American Association for Cancer Research (AACR).  When he is not on the bench you will find him spending time with his family or exploring the world through traveling and blogging.

Cover image: (Cell Image Library) Color-enhanced scanning electron micrograph of the inside of a guinea pig inner ear showing the hearing organ, or cochlea. Running along the spiral structure are rows of sensory cells which respond to different frequencies of sound. The whole organ is just a few millimeters long.

The contents of Club SciWri are the copyright of PhD Career Support Group for STEM PhDs {A US Non-Profit 501(c)3}. (PhDCSG is an initiative of the alumni of the Indian Institute of Science, Bangalore. The primary aim of this group is to build a NETWORK among scientists, engineers and entrepreneurs).

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Disclaimer: The authors and editors for Medness Plus declare no financial benefits or remuneration from the sponsors. The sponsorships support the non-profit organization PhD Career Support Group (PhD CSG). The research conducted by authors and editors is a voluntary effort to popularize science for the public on behalf of PhD CSG. The sponsors do not have any influence on the nature or kind of the news/analysis reported in Onco-this-Week. The views and opinions expressed in this article are those of the authors and do not necessarily reflect the official policy or position of Club SciWri or PhD CSG. Examples of analysis performed within this article are only examples. They should not be utilized in real-world analytic products as they are based only on very limited and dated open source information. Assumptions made within the analysis are not reflective of the position of anyone volunteering or working for Club SciWri or PhD CSG. This blog is strictly for news and information. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

 

 

 

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The contents of Club SciWri are the copyright of Ph.D. Career Support Group for STEM PhDs (A US Non-Profit 501(c)3, PhDCSG is an initiative of the alumni of the Indian Institute of Science, Bangalore. The primary aim of this group is to build a NETWORK among scientists, engineers, and entrepreneurs).

This work by Club SciWri is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.

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