FDA grants approval for the first test to help in detecting cytomegalovirus, a type of herpes virus in newborns
FDA recently authorized the marketing of a new diagnostic test, the Alethia CMV Assay Test System, to detect a cytomegalovirus (CMV), a type of herpes virus, in newborns less than 21 days of age [1]. The test system was authorized to Meridian Bioscience, Inc.
Meridian Bioscience, Inc., Stock (VIVO) in Spot Light Today After FDA Granted Marketing Authorization of Alethia CMV Assay Test System to the Company https://t.co/hOSRos4F5j pic.twitter.com/LuhMzVBzi4
— InstitutionalTrading (@mrstardom) December 3, 2018
Based on statistics from the Center for Disease Control and Prevention, approximately, by the age of 40, about half the adults are infected by CMV. Even though no specific signs or symptoms are associated with it, the infection by CMV can be detrimental in patients with a weakened immune system and in case of certain newborns. Congenital CMV occurs when a baby is infected with the virus during pregnancy. No symptoms or signs are exhibited but the baby can develop serious long-term health and hearing issues. The congenital CMV can now be detected using the Alethia CMV Assay test. It is quite simple as saliva swab taken from a newborn is tested for CMV deoxyribonucleic acid (DNA). The test results are recommended to be used along with other clinical information and diagnostic tests to make a more informed decision about the next course of action. The authorization was issued based on data from the review of clinical and analytical performance of the device. A clinical study data showed that out of 1,475 saliva samples collected from newborns, 1,472 were correctly identified by the device as negative for the CMVDNA. Three of them were incorrectly identified to be positive when they were negative.Five samples were correctly identified to be positive for the CMV DNA. Further tests were also done on 34 archived samples of newborns who were infected withCMV. All 34 of them came out positive when detected for CMV DNA by using the device. This set of data was also taken into consideration by FDA while reviewing the candidature of the device as a diagnostic test to detect the cytomegalovirus.FDA reviewed the test system through the de novo premarket review pathway, a regulatory pathway for novel, low-to-moderate-risk devices of a new type. A new criterion of special controls is being established by the FDA which will determine the requirements to depict the effectiveness, reliability and accuracy of the tests that claim to be used as a diagnostic test for detectingCMV in conjunction with other clinical data. A fulfillment of these special controls along with the general ones is expected to provide a good estimate about the safety and efficacy of such tests. A new regulatory classification was also created in the process. This means that any new device that has the same typeof intended use will go through FDA’s 510(k) process based on which, marketing authorization of the device will be obtained by demonstrating substantial equivalence to a previously authorized device.
From Visually.
“Although most people who become infected with cytomegalovirus face little to no risk of serious illness, the virus has the potential to cause serious illness for people with weak immune systems and in newborn babies,” said Tim Stenzel, director of the Office of In Vitro Diagnostics and Radiological Health in the FDA’s Center for Devices and Radiological Health. “This test for detecting the virus, when used in conjunction with the results of other diagnostic tests, may help health care providers more quickly identify the virus in newborns and determine the best approach for the child.”
FDA approves Firdapse tablets for the treatment of a rare autoimmune disorder, Lambert-Eaton myasthenic syndrome.
FDA recently announced the first treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults [2]. The organization announced the use of Firdapse (amifampridine) tablets for the treatment of this rare autoimmune disorder that affects the connection between muscles and nerves causing weakness and other symptoms in affected patients.The approval was granted to Catalyst Pharmaceuticals Inc.
After 2016 Rejection, Catalyst Wins Approval for LEMS Treatment Firdapse https://t.co/Vq6cLF0nIF #pharma pic.twitter.com/IG1dcDvnD6
— Rochelle St. James (@MedSchoolGuru) December 13, 2018
LEMS is an autoimmune disorder where the body’s own immune system attacks the connections between the nerves and the muscles (neuromuscular junction) and disrupts the signaling pathway between the nerve and muscle cells. LEMS is associated with various other autoimmune diseases. But very often, it occurs in patients with small cell lung cancer where its onset will either precede or coincide with the diagnosis of cancer. Based on the available data, LEMS is prevalent in three million people worldwide. Two clinical trials were taken into consideration while determining the efficiency of Firdapse. The studies involved 64 adult patients receiving the drug and placebo. Subject Global Impression is a seven-point scale on which the patients rate their overall impression of their well-being after the study treatment. Quantitative Myasthenia Gravis score is a 13-item physician-rated scale assessing the muscular weakness. Both these measuring parameters were taken into consideration when the assessment of the efficacy of the drug was being made. In both cases, the patients receiving the drug benefitted more than those who were on placebo. Firdapse has received a Priority Review,Breakthrough Therapy and Orphan Drug designation that will enhance the research of drugs to treat rare diseases. The most common side effects experienced by the patients during the clinical trials were upper respiratory tract infection, nausea, abdominal pain, burning or pricking sensation (paresthesia), elevated liver enzymes, diarrhea, hypertension, back pain, muscle spasms and headache.In some cases, with no prior history, patients were seen to develop seizures. A warning has been issued to inform the healthcare provider, in case any patients experience rash, hives, hypersensitivity, swelling or have trouble breathing.
From Visually.
“There has been a long-standing need for a treatment for this rare disorder,” said Billy Dunn, M.D., director of the Division of Neurology Products in the FDA’s Center forDrug Evaluation and Research. “Patients with LEMS have significant weakness and fatigue that can often cause great difficulties with daily activities.”
FDA approves the ACTpen for Genentech’s ACTEMRA, for the treatment of Giant Cell Arteritis, Rheumatoid Arthritis and two other forms of Juvenile Arthritis
Genentech recently announced the approval of ACTPen 162/0.9 mL, single-dose pre-filled auto injector for Actemra (tocilizumab) [3]. It will serve as an additional formulation for adult patients with moderate-to-severe active rheumatoid arthritis, with patients with giant cell arteritis (GCA) and for those who have inadequate response to one or more anti-rheumatic drugs (DMARDs). It can also be administered by caregivers to patients two years and older with active systemic juvenile idiopathic arthritis (SJIA) and active polyarticular juvenile idiopathic arthritis (PJIA). Self-injection of pediatric patients is yet to be tested. ACTPen is scheduled to be available in January 2019.
Rheumatoid arthritis is a very common autoimmune disease and is known to impact 1.5million people in the United States with around three-quarters being prevalent in women. The disease is progressive in nature and is mainly associated with pain and swelling in and around the joint tissues. Irreversible and detrimental effects will be experienced by patients, if the condition is left untreated.Temporal arteritis or giant cell arteritis is known to affect around 228,000people over the age of 50 in the U.S. and just like rheumatoid arthritis, GCA is also likely to affect more women than men. If left untreated, it can cause stroke, blindness and aortic aneurysms along with the usual symptoms of headache and jaw pain. PJIA and SJIA are forms of juvenile idiopathic arthritis (JIA), a chronic arthritis form affecting children. Among 300,000 children in the U.S. affected by JIA, PJIA accounts for 25 percent and SJIA accounts for around 10 percent. SJIA is usually characterized by inflammation in one or multiple joints. There may also be fever for at least two weeks accompanied with skin rash. Inflammation of the lining of lungs and/or heart, enlargement of liver and spleen and anemia are among some of the other symptoms in case of people with SJIA. Those detected with PJIA show inflammation in five or more joints in parts of the body such as hands and feet and are visible within the first six months of the disease. Actemra intravenous infusion formulation (IV) for adults was first approved by the FDA in January 2010 for treating rheumatoid arthritis. Actemra’s pre-filled syringe (PFS) formulations for the subcutaneous injection (SC) for adults with RA, was approved in October 2013. There were a few more FDA approvals for Actemra between 2010 and 2017 which resulted in its use for the treatment of one million patients in this period. Actemra SC was approved by the FDA for the treatment of GCA in May 2017. Actemra IV was approved in April 2011 and April 2013 for patients two years and older with active SJIA and active PJIA respectively. Actemra is the first humanized IL-6 (interleukin-6) receptor antagonist approved for the treatment of adults with moderate-to-severe RA and those who show inadequate response to disease modifying anti rheumatic drugs. Only healthcare practitioners are licensed to treat people with Actemra. FDA’s approval of ACTPen was based on the clinical data from two studies. The first one was a randomized, open-label, two-period, crossover Phase I study, involving 188 healthy volunteers thus investigating the relative bioavailability of one injection of Actemra 162 mg SC via PFS with needle safety device to a single injection of the same Actemra SC via ACTPen.The second trial was a non-randomized, open-label, observational Phase IV human factors study involving 54 adult RA patients to investigate efficacy and safety of the ACTPen administration by self, caregivers or healthcare professionals.The data revealed that single-dose SC administration of 162 mg Actemra with ACTPen was bio equivalent to the administration with the PFS and the intended users successfully did the administration.
From Visually.
“When it comes to the administration of medicines, we believe patients should have choices, when possible,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “With ACTPen for Actemra, we are pleased to offer an additional option to patients who may prefer using the new auto injector over other formulations.”
FDA approves DEXTENZA, a product of Ocular Therapeutix, for the treatment of Ocular pain after an Ophthalmic surgery
FDA recently announced the approval of DEXTENZA (dexamethasone ophthalmic insert) for the intra canalicular insert for the treatment of ocular pain after an ophthalmic surgery and it is the first of its kind [4]. The approval was given to Ocular Therapeutix Inc., a biopharmaceutical company that specializes in the development and marketing of unique therapies for various diseases and conditions of the eye.
DEXTENZA is a corticosteroid that is used for the treatment of ocular pain after ophthalmic surgery. It is a preservative-free ophthalmic insert, inserted to the lower lacrimal punctum and into the canaliculus. A single DEXTENZA is known to release a 0.4 mg dose of dexamethasone till up to30 days after the insertion. It does not require removal since it can easily be reabsorbed. DEXTENZA is implicated for single-use only. The approval was based on the data from two randomized, double-masked, multicenter, vehicle controlled, parallel group Phase 3 clinical trials. Immediately after cataract surgery, the patients received DEXTENZA or its vehicle. In the first study, at day 8, 80% of those treated with DEXTENZA were pain-free as compared to 43% of those who were vehicle-treated. In the second study, at day 8, 77% of those treated with DEXTENZA were pain-free when compared to 59% of the vehicle-treated ones. A total of 351 subjects were subjected to DEXTENZA andthe most common ocular adverse effects experienced included anterior chamber inflammation, iritis, iridocyclitis, reduced visual sharpness, cystoid macular edema, increased intraocular pressure, eye pain and conjunctival hyperemia.Headache was the most common non-ocular adverse effect. DEXTENZA will offer patients a convenient full course of post-surgical steroid treatment after a single intracanalicular insert by the physician. The company thus hopes to develop this potentially transformational new product for patients and physicians.
“We are extremely pleased to announce the approval of DEXTENZA, coming so soon after our pre-approval inspection and approximately one month ahead of the PDUFAdate,” said Antony Mattessich, the Company’s President and Chief ExecutiveOfficer. “Just over a year ago, we set out to augment our scientific and formulation expertise with individuals who have the skills and experience to create a first-class team to get DEXTENZA approved and become a commercial stage biopharmaceutical company. We believe this approval is a major external validation of the drug delivery technology platform, and of the transformation that has taken place at Ocular. While we are excited by the approval of our first drug product, our goal has always been to bring DEXTENZA to as many patients as possible in the near term and to revolutionize ophthalmic drug delivery by making drops obsolete. We now turn our efforts towards the successful commercial launch of DEXTENZA.” “Compliance with taking eye drops after eye surgery is very challenging for patients and a concern for surgeons,” said Michael Goldstein, MD, Chief Medical Officer. “The approval of DEXTENZA offers surgeons the opportunity to treat patients with a preservative-free steroid after surgery with the placement of a single drug insert. With this product, patients may be liberated from having to deal with the burdensome regimen of using steroid eye drops after ophthalmic surgery.”
FDA announces the clearance of reSET-O™, the first Prescription Digital Therapeutic(PDT), for the treatment of opioid use disorder
Pear Therapeutics and Sandoz Inc, a division of Novartis, recently announced that they have received FDA clearance for reSET-OTM. It is the firstFDA-cleared Prescription Digital Therapeutic (PDT) for patients with opioid use disorder [5]. This will be a great addition to the digital revolution ofNovartis and will add on to the initiative taken by Sandoz to improve access to treatment through a digital platform and thus increase the engagement of patients.
Opioid Use Disorder (OUD) involves the addiction and misuse of opioids including prescription medications such as pain relievers, and synthetic opioids. It is at present a serious national crisis in which approximately 115 Americans die each day from overdosing. The economic burden associated with it is huge as well, considering the costs of healthcare, addiction treatment and criminal justice involvement. Relapse rates and high attrition are the foremost problems intreating patients with OUD. Thus, the success of a treatment outcome in the case of OUD patients would be evaluated based on retention of patients in the treatment. reSET-O aims at retention of OUD patients in outpatient treatment by providing cognitive behavioral therapy (CBT) in addition to outpatient treatment that includes transmucosal buprenorphine (medication assisted treatment, MAT) and contingency management, for patients 18 years and older and are currently under supervision. reSET-O is a prescription-only mobile medical application. This is a 12-week interval PDT for OUD. It is engineered to deliver CBT to such patients and is based on the Community ReinforcementApproach (CRA). The CRA therapy is delivered in the form of interactive therapy lessons by reSET-O. Cognitive behavioral therapy component and skill-building exercises are the constituents of each therapy lesson, which in turn is in the form of audio, text or video, animations and graphics. reSET-O is an effective way for patients to maintain communication with their clinician by reporting cravings and triggers. Based on the deal for marketing, Sandoz will be the leader for commercialization of reSET-O and reSET, Pear’s PDT for the treatment of Substance Use Disorder. It is an adjunct to the standard care offered to OUD patients. A National Institute on Drug Abuse, sponsored a clinical trial to support the FDA submission. This included evaluation of the therapeutic in 170patients with OUD in 12 weeks. The patients were randomly subjected to one or the other form of treatment. One was the usual treatment with standard clinician interactions along with buprenorphine and the other was reSET-O with the standard clinician interaction in conjunction with buprenorphine. From the data it was evident that patients randomized to reSET-O CBT along with the outpatient treatment and contingency management showed a much higher retention as compared to the other treatment.
From Visually.
“Nearly 50,000 drug overdose deaths involving opioids, including prescription pain medications and heroin, took place in the U.S. in 2017,” said CoreyMcCann, M.D., Ph.D., President and CEO of Pear Therapeutics. “There is an urgent need for new and innovative therapeutics to address this public health epidemic. This groundbreaking decision by the FDA ushers in a new standard for treating patients with Opioid Use Disorder and it signals a new path for therapeutic software to be used in conjunction with pharmacotherapy to improve efficacy.” “Digital technologies and data science have incredible potential to unlock the next chapter of medical innovation and to help individuals finally take control of their own health in a meaningful way,” saidRichard Francis, CEO, Sandoz. “New digital therapeutics such as reSET-O also have the potential to fundamentally change how patients interact with their therapies and thus improve patient outcomes. At Sandoz, we are proud to be a joint pioneer in this exciting new field.”
Tris Pharma recalls infants’ ibuprofen due to potential higher concentrations of the drug
Tris Pharma, Inc. recently recalled three lots of its infant Ibuprofen concentrated oral suspension, USP (NSAID) 50 mg per 1.25 mL to the retail level [6]. The reason for the recall is that these lots of the product have been found to contain higher concentrations of ibuprofen.
Tris Pharma is an integrated pharmaceutical company that mainly focusses on the development of innovative medications to satisfy the unfulfilled needs of patients. They have used their technology platform, LiquiXR to deliver sustained release in the form of liquid, chewable, orally disintegrating tablets that are useful to patients. Very recently Tris Pharma sold the erroneous lot of Ibuprofen Concentrated Oral Suspension, USP (NSAID) 50 mg per 1.25 mL to one customer which distributed the lot into the US market. This product is used as fever reducer and pain reliever and has been packaged in 0.5 oz bottles. An urgent recall notice has been sent to the customer and a return is being arranged for the product. Wholesalers and retailers of the product have been advised to stop the further distribution of the lot and a detailed list has been issued by the company to help them return the product. No adverse effects have yet been reported related to this product lot. But there is a possibility that some infants who are more susceptible to a higher concentration of drug can be vulnerable to NSAID-associated renal injury. Some of the adverse effects associated include vomiting, nausea, diarrhea, tinnitus, headache and gastrointestinal bleeding.
Eli Lilly and AC Immune SA sign a collaboration agreement to extend their research in neurodegeneration
Eli Lilly and AC Immune recently announced that they have entered into a collaboration by signing a license agreement to research and develop tau aggregation inhibitor small molecule for the treatment of neurodegenerative diseases such as Alzheimer’s [7]. AC Immune’s lead molecule, ACI-3024, which has already demonstrated tau aggregation inhibition in preclinical model, will be the primary focus of the collaboration.
For selectively and effectively reducing toxic intracellular misfolded and aggregated tau, a series of chemically small molecules (Morphomers) have been identified. Targeting misfolded and aggregated tau is the most potent method to control the spread of tau pathology throughout the brain,. A pathological feature of Alzheimer’s disease, is the associated neuroinflammatory markers. A reduction in these markers signifies a reduction in tau pathology. Based on the terms of the agreement, an upfront payment will be made to AC Immune of CHF80 million and $50 million in exchange for a note convertible to equity at a premium. AC immune will also be eligible to receiveCHF60 million as near-term development milestones along with other commercial and regulatory ones to an approximate CHF1.7 billion and tiered royalty payments. The initial Phase 1 trial will be conducted by AC Immune involving the development of the Morphomer tau aggregation inhibitors. Lilly will fund and conduct further developments. Lilly is supposed to receive the worldwide commercialization rights for the tau aggregation inhibitors for Alzheimer’s. Certain development rights in orphan indications and co-development and co-promotion options in certain diseases outside the Alzheimer’s, has been retained by AC Immune. According to the Generally Accepted AccountingPrinciples (GAAP), the transactions will be reflected in Lilly’s reported results and financial guidance. No change will be made to Lilly’s 2018 non-GAAP earnings per share guidance due to this transaction. The transaction is subjected to clearance under certain customary closing conditions and under Hart-Scott-Rodino Antitrust Improvements Act.
From Visually.
“This landmark partnership with Lilly is transformational for the future of ACImmune. Lilly’s substantial experience in neurology, and particularly inAlzheimer’s disease, is a major validation of our small-molecule platform forCNS therapeutics. It also demonstrates the potential of our pre-clinical assets and adds substantial value to our pipeline. We look forward to working closely with Lilly in this exciting field over the coming years,” said Prof. Andrea Pfeifer, CEO of AC Immune. “Lilly is an industry leader in Alzheimer’s research, with numerous ongoing scientific programs that target suspected causes of the disease, including amyloid plaques and tau tangles,” said Mark Mintun, M.D., vice president of neurodegeneration and pain research at Lilly.” This agreement with AC Immune represents another opportunity to hopefully make progress against this devastating disease, and we look forward to together bringing tau aggregation inhibitors into clinical development.”
Reference:
[1] https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm627310.htm.
[2] https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm627093.htm.
[3]https://www.gene.com/media/press-releases/14767/2018-11-26/fda-approves-the-actpen-for-genentechs-a.
[7] http://cws.huginonline.com/A/171580/PR/201812/2228775_5.html.
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 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) Description: “Confocal micrograph of a mast cell in human eye with conjunctivitis. This image shows a single mast cell invading conjunctival tissue in response to an inflammatory agent or pathogen. The mast cell contains vesicles of histamine (red dots). Mast cells are among the first cells of the immune system to react to the presence of an invading pathogen and they facilitate the movement of leukocytes (white blood cells) and other immune cells toward the site of infection. The image is composed of 42 stacked sections. Honorable Mention, 2011 Olympus BioScapes Digital Imaging Competition®.”
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