Flibanserin

Presentations showcase diversity and momentum of Pfizer’s growing portfolio of vaccines and anti-infectives NEW YORK--(BUSINESS WIRE)-- Pfizer Inc. (NYSE: PFE) will present data across its infectious disease portfolio at the upcoming IDWeek 2023 held in Boston from October 11-15, 2023. Data from 45 abstracts will highlight the advances Pfizer is making in helping prevent and treat certain infectious diseases, particularly respiratory illnesses. This will include research featured in the IDWeek press program assessing the potential public health impact of ABRYSVO™ (Respiratory Syncytial Virus Vaccine), the company’s bivalent respiratory syncytial virus prefusion F (RSVpreF) vaccine for maternal immunization to protect infants against RSV illness. “The data at this year’s IDWeek highlight Pfizer’s commitment to transforming respiratory health, an area where we are proud to have delivered several innovative vaccine and treatment options over the last three years. We also continue to actively investigate infectious diseases more broadly, with a diverse pipeline of innovative vaccine and anti-infective treatment candidates,” said Annaliesa Anderson, Ph.D., Senior Vice President and Head, Vaccine Research and Development, Pfizer. “With our unique scientific capabilities and vaccine technology platforms, we are unwavering in the fight against the world’s most pressing disease challenges. We are excited to present our latest clinical results that support our growing infectious disease portfolio for the purpose of improving global health outcomes.” "The burden of respiratory illnesses on global public health is significant. It is particularly relevant as we enter the winter respiratory season in the Northern Hemisphere and once again face the threat of a possible tripledemic with influenza, RSV and COVID-19 co-circulating,” said Luis Jodar, Ph. D., Chief Medical Affairs Officer, Vaccines/Antivirals and Evidence Generation, Pfizer. “At IDWeek, we are pleased to share real-world data that will help educate healthcare professionals about the role Pfizer’s vaccines and treatments can play in helping mitigate the impact of certain respiratory infections during this critical period.” Pfizer's participation at IDWeek 2023 comprises breakthrough research from across the company’s robust infectious disease portfolio, covering RSV, COVID-19, pneumococcal disease, Lyme disease, meningococcal disease, multidrug-resistant gram-negative bacterial infections and more. Eight abstracts have been selected for oral presentation, including data on the successful coadministration of ABRYSVO with an influenza vaccine in older adults and results from the Phase 3 REVISIT study of Pfizer’s investigational antibiotic combination aztreonam-avibactam (ATM-AVI) candidate. Data highlighting the real-world effectiveness of Pfizer’s FDA-approved COVID-19 oral treatment for adults, PAXLOVID™ (nirmatrelvir tablets; ritonavir tablets), will also be featured at the meeting. A complete list of Pfizer-sponsored accepted abstracts is available here. Details for Pfizer-sponsored oral presentations are below: Title/ Abstract Number Presenting Name/Type Date/ Time EDT Location IDWEEK PRESS PROGRAM 1942 - Potential Public Health Impact of Bivalent Respiratory Syncytial Virus Prefusion F (RSVpreF) Maternal Vaccine for Prevention of RSV Among US Infants Amy W. Law, PharmD Oct 13 2:15 - 2:30 PM US ET 104 ABC ORAL PRESENTATIONS 1019 - Respiratory Syncytial Virus (RSV) Diagnoses in Hospitalized Patients Increases when Additional Specimen Types are Added to Nasopharyngeal Swab: Results from the RSV in Adults Multispecimen Study Elizabeth Begier, MD, MPH Oct 12 3:15 - 3:33 PM US ET 104 ABC 1941 - Coadministration of Bivalent Respiratory Syncytial Virus (RSVpreF) Vaccine with Influenza Vaccine in Older Adults James Baber, MBChB, MPH Oct 13 2:00 - 2:15 PM US ET 104 ABC 1944 - Tolerability and Safety of 20-Valent Pneumococcal Conjugate Vaccine (PCV20) in Infants and Older Children in Global Studies Kathleen McElwee, MD, MPH Oct 13 2:45 - 3:00 PM US ET 104 ABC 1948 - Rise of Candida auris Infections Worldwide and Trends on the Activity of Fosmanogepix and Comparator Agents Against C. auris Causing Invasive Infections Cecilia G. Carvalhaes, MD, PhD Oct 13 2:30 - 2:45 PM US ET 102 AB 1949 – Activity of Aztreonam-Avibactam Against Enterobacterales Resistant to Recently Approved Beta-Lactamase Inhibitor Combinations Collected Worldwide (ex-US: 2020-2022) Helio S. Sader, MD, PhD, FIDSA Oct 13 2:45 - 3:00 PM US ET 102 AB 2890 - Pharmacokinetics, Safety, and Efficacy of Ceftazidime-Avibactam in Neonates and Young Infants with Bacterial Infections: Results from a Phase 2a, 2-part, Open-label, Non-randomized, Multicenter Trial Richard D England, MD Oct 14 2:05 - 2:15 PM US ET 254 AB 2893 - Efficacy and Safety of Aztreonam-Avibactam for the Treatment of Serious Infections Due to Gram-Negative Bacteria, Including Metallo-β-Lactamase-Producing Pathogens: Phase 3 REVISIT Study Yehuda Carmeli, MD MPH Oct 14 2:35 - 2:45 PM US ET 254 AB INDICATIONS AND IMPORTANT SAFETY INFORMATION FOR ABRYSVO U.S. INDICATIONS ABRYSVOTM is a vaccine indicated for: the prevention of lower respiratory tract disease (LRTD) caused by respiratory syncytial virus (RSV) in people 60 years of age and older pregnant individuals at 32 through 36 weeks gestational age for the prevention of LRTD and severe LRTD caused by RSV in infants from birth through 6 months of age IMPORTANT SAFETY INFORMATION ABRYSVO should not be given to anyone with a history of severe allergic reaction (eg, anaphylaxis) to any of its components For pregnant individuals: to avoid the potential risks of preterm birth, ABRYSVO should be given during 32 through 36 weeks gestational age Fainting can happen after getting injectable vaccines, including ABRYSVO. Precautions should be taken to avoid falling and injury during fainting Adults with weakened immune systems, including those receiving medicines that suppress the immune system, may have a reduced immune response to ABRYSVO Vaccination with ABRYSVO may not protect all people In adults 60 years of age and older, the most common side effects (≥10%) were fatigue, headache, pain at the injection site, and muscle pain In pregnant individuals, the most common side effects (≥10%) were pain at the injection site, headache, muscle pain, nausea In clinical trials where ABRYSVO was compared to placebo, infants born to pregnant individuals experienced low birth weight (5.1% ABRYSVO versus 4.4% placebo) and jaundice (7.2% ABRYSVO versus 6.7% placebo View the full Prescribing Information. INDICATION, AUTHORIZED USE AND IMPORTANT SAFETY INFORMATION FOR PAXLOVID U.S. Indication PAXLOVID is indicated for the treatment of mild-to-moderate coronavirus disease 2019 (COVID-19) in adults who are at high risk for progression to severe COVID-19, including hospitalization or death. Limitations of Use PAXLOVID is not approved for use as pre-exposure or post-exposure prophylaxis for prevention of COVID-19. U.S. FDA Emergency Use Authorization Statement The U.S. Food and Drug Administration (FDA) has issued an Emergency Use Authorization (EUA) for the emergency use of PAXLOVID for the treatment of adults and pediatric patients (12 years of age and older weighing at least 40 kg) with mild to moderate coronavirus disease 2019 (COVID-19) and who are at high risk for progression to severe COVID-19, including hospitalization or death. PAXLOVID has not been approved, but has been authorized for emergency use by FDA under an EUA, for the treatment of mild-to-moderate COVID-19 in pediatric patients (12 years of age and older weighing at least 40 kg) who are at high risk for progression to severe COVID-19, including hospitalization or death. The emergency use of PAXLOVID is only authorized for the duration of the declaration that circumstances exist justifying the authorization of the emergency use of drugs and biological products during the COVID-19 pandemic under Section 564(b)(1) of the Act, 21 U.S.C. § 360bbb-3(b)(1), unless the declaration is terminated or authorization revoked sooner. IMPORTANT SAFETY INFORMATION WARNING: SIGNIFICANT DRUG INTERACTIONS WITH PAXLOVID PAXLOVID includes ritonavir, a strong CYP3A inhibitor, which may lead to greater exposure of certain concomitant medications, resulting in potentially severe, life-threatening, or fatal events Prior to prescribing PAXLOVID: 1) Review all medications taken by the patient to assess potential drug-drug interactions with a strong CYP3A inhibitor like PAXLOVID and 2) Determine if concomitant medications require a dose adjustment, interruption, and/or additional monitoring Consider the benefit of PAXLOVID treatment in reducing hospitalization and death, and whether the risk of potential drug-drug interactions for an individual patient can be appropriately managed PAXLOVID is contraindicated in patients with a history of clinically significant hypersensitivity reactions (eg, toxic epidermal necrolysis or Stevens-Johnson syndrome) to its active ingredients (nirmatrelvir or ritonavir) or any other components of the product. If signs and symptoms of a clinically significant hypersensitivity reaction or anaphylaxis occur, immediately discontinue PAXLOVID and initiate appropriate medications and/or supportive care. PAXLOVID is contraindicated with drugs that are primarily metabolized by CYP3A and for which elevated concentrations are associated with serious and/or life-threatening reactions and drugs that are strong CYP3A inducers where significantly reduced nirmatrelvir or ritonavir plasma concentrations may be associated with the potential for loss of virologic response and possible resistance. There are certain other drugs for which concomitant use with PAXLOVID should be avoided and/or dose adjustment, interruption, or therapeutic monitoring is recommended. Drugs listed here are a guide and not considered a comprehensive list of all drugs that may be contraindicated with PAXLOVID. The healthcare provider should consult other appropriate resources such as the prescribing information for the interacting drug for comprehensive information on dosing or monitoring with concomitant use of a strong CYP3A inhibitor like PAXLOVID. Drugs that are primarily metabolized by CYP3A for which elevated concentrations are associated with serious and/or life-threatening reactions: Alpha 1-adrenoreceptor antagonist: alfuzosin Antianginal: ranolazine Antiarrhythmic: amiodarone, dronedarone, flecainide, propafenone, quinidine Anti-gout: colchicine (in patients with renal and/or hepatic impairment) Antipsychotics: lurasidone, pimozide Benign prostatic hyperplasia agents: silodosin Cardiovascular agents: eplerenone, ivabradine Ergot derivatives: dihydroergotamine, ergotamine, methylergonovine HMG-CoA reductase inhibitors: lovastatin, simvastatin (these drugs can be temporarily discontinued to allow PAXLOVID use) Immunosuppressants: voclosporin Microsomal triglyceride transfer protein inhibitor: lomitapide Migraine medications: eletriptan, ubrogepant Mineralocorticoid receptor antagonists: finerenone Opioid antagonists: naloxegol PDE5 inhibitor: sildenafil (Revatio®) when used for pulmonary arterial hypertension Sedative/hypnotics: triazolam, oral midazolam Serotonin receptor 1A agonist/serotonin receptor 2A antagonist: flibanserin Vasopressin receptor antagonists: tolvaptan Drugs that are strong CYP3A inducers: PAXLOVID cannot be started immediately after discontinuation of any of the following medications due to the delayed offset of the recently discontinued CYP3A inducer: Anticancer drugs: apalutamide Anticonvulsant: carbamazepine, phenobarbital, primidone, phenytoin Antimycobacterials: rifampin, rifapentine Cystic fibrosis transmembrane conductance regulator potentiators: lumacaftor/ivacaftor Herbal Products: St. John’s Wort (hypericum perforatum) Risk of Serious Adverse Reactions Due to Drug Interactions: Initiation of PAXLOVID, which contains ritonavir, a strong CYP3A inhibitor, in patients receiving medications metabolized by CYP3A or initiation of medications metabolized by CYP3A in patients already receiving PAXLOVID, may increase plasma concentrations of medications metabolized by CYP3A. Medications that induce CYP3A may decrease concentrations of PAXLOVID. These interactions may lead to: Clinically significant adverse reactions, potentially leading to severe, life-threatening, or fatal events from greater exposures of concomitant medications Loss of therapeutic effect of PAXLOVID and possible development of viral resistance Severe, life-threatening, and/or fatal adverse reactions due to drug interactions have been reported in patients treated with PAXLOVID. The most commonly reported concomitant medications resulting in serious adverse reactions were calcineurin inhibitors (eg, tacrolimus, cyclosporine), followed by calcium channel blockers. Hepatotoxicity: Hepatic transaminase elevations, clinical hepatitis, and jaundice have occurred in patients receiving ritonavir. Therefore, caution should be exercised when administering PAXLOVID to patients with pre-existing liver diseases, liver enzyme abnormalities, or hepatitis. Because nirmatrelvir is coadministered with ritonavir, there may be a risk of HIV-1 developing resistance to HIV protease inhibitors in individuals with uncontrolled or undiagnosed HIV-1 infection. The most common adverse reactions in the PAXLOVID group (≥1%) that occurred at a greater frequency than in the placebo group were dysgeusia (5% and <1%, respectively) and diarrhea (3% and 2%, respectively). The following adverse reactions have been identified during use of PAXLOVID under Emergency Use Authorization: Immune System Disorders: Anaphylaxis, hypersensitivity reactions Skin and Subcutaneous Tissue Disorders: Toxic epidermal necrolysis, Stevens-Johnson syndrome Nervous System Disorders: Headache Vascular Disorders: Hypertension Gastrointestinal Disorders: Abdominal pain, nausea, vomiting General Disorders and Administration Site Conditions: Malaise PAXLOVID is a strong inhibitor of CYP3A, and an inhibitor of CYP2D6, P-gp, and OATP1B1. Coadministration of PAXLOVID with drugs that are primarily metabolized by CYP3A and CYP2D6 or are transported by P-gp or OATP1B1 may result in increased plasma concentrations of such drugs and increase the risk of adverse events. Coadministration with other CYP3A substrates may require a dose adjustment or additional monitoring. Pregnancy: Available data on the use of nirmatrelvir during pregnancy are insufficient to evaluate for a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. Published observational studies on ritonavir use in pregnant women have not identified an increase in the risk of major birth defects. Published studies with ritonavir are insufficient to identify a drug-associated risk of miscarriage. There are maternal and fetal risks associated with untreated COVID-19 in pregnancy. Lactation: There are no available data on the presence of nirmatrelvir in human or animal milk, the effects on the breastfed infant, or the effects on milk production. A transient decrease in body weight was observed in the nursing offspring of rats administered nirmatrelvir. Limited published data report that ritonavir is present in human milk. There is no information on the effects of ritonavir on the breastfed infant or the effects of the drug on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for PAXLOVID and any potential adverse effects on the breastfed infant from PAXLOVID or from the underlying maternal condition. Contraception: Use of ritonavir may reduce the efficacy of combined hormonal contraceptives. Advise patients using combined hormonal contraceptives to use an effective alternative contraceptive method or an additional barrier method of contraception. Pediatrics: The optimal dose of PAXLOVID has not been established in pediatric patients. Systemic exposure of nirmatrelvir increases in renally impaired patients with increase in the severity of renal impairment. No dosage adjustment is recommended in patients with mild renal impairment. Reduce the dose of PAXLOVID in patients with moderate renal impairment (eGFR ≥30 to <60 mL/min). PAXLOVID is not recommended in patients with severe renal impairment (eGFR <30 mL/min) or in patients with end-stage renal disease (eGFR <15 mL/min). PAXLOVID is not recommended for use in patients with severe hepatic impairment (Child-Pugh Class C). Please see Full Prescribing Information, including BOXED WARNING and Patient Information Click for Fact Sheets: For Consumers: EUA Fact sheet for Patients, Parents, and Caregivers For Healthcare Professionals: EUA Fact Sheet for HCPs INDICATIONS AND IMPORTANT SAFETY INFORMATION FOR PREVNAR 20 U.S. INDICATIONS PREVNAR 20® is a vaccine approved for: the prevention of invasive disease caused by 20 Streptococcus pneumoniae strains (1, 3, 4, 5, 6A, 6B, 7F, 8, 9V, 10A, 11A, 12F, 14, 15B, 18C, 19A, 19F, 22F, 23F, and 33F) in individuals 6 weeks and older. the prevention of otitis media (middle ear infection) caused by 7 of the 20 strains in individuals 6 weeks through 5 years. IMPORTANT SAFETY INFORMATION PREVNAR 20 should not be given to anyone who has had a severe allergic reaction to any component of PREVNAR 20 or to diphtheria–toxoid-containing vaccine. Individuals with weakened immune systems may have a lower immune response. Safety data are not available for these groups. A temporary pause in breathing after getting the vaccine has been observed in some infants who were born prematurely. For premature infants, talk to your doctor about the infant's medical status when deciding to get vaccinated with PREVNAR 20. In individuals 2, 4, 6, and 12 through 15 months of age vaccinated with a 4-dose schedule, the most common side effects reported at a rate of >10% were irritability, pain at the injection site, drowsiness, decreased appetite and injection site redness, injection site swelling, and fever. In individuals 15 months through 17 years of age vaccinated with a single dose, the most common side effects reported at a rate of >10% were irritability, pain at the injection site, drowsiness, fatigue and muscle pain, decreased appetite, injection site swelling and injection site redness, headache, and fever. Ask your doctor about the risks and benefits of PREVNAR 20. Only a doctor can decide if PREVNAR 20 is right for your child. View the full Prescribing Information. About Pfizer: Breakthroughs That Change Patients’ Lives At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at . In addition, to learn more, please visit us on and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer. Disclosure Notice The information contained in this release is as of October 5, 2023. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments. This release contains forward-looking information about Pfizer’s infectious disease pipeline, in-line products and product candidates, including ABRYSVO (RSVpreF), Pfizer’s aztreonam-avibactam (ATM-AVI) candidate and PAXLOVID, including their potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, uncertainties regarding the commercial success of ABRYSVO (RSVpreF), Pfizer’s ATM-AVI candidate, PAXLOVID or any of Pfizer’s other infectious disease products or product candidates; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as risks associated with pre-clinical and clinical data (including Phase 1/2/3 or Phase 4 or pre-clinical data for ABRYSVO (RSVpreF), Pfizer’s ATM-AVI candidate, PAXLOVID or any of Pfizer’s other infectious disease products or product candidates, including the data discussed in this release) in any of our studies in pediatrics, adolescents, or adults or real world evidence, including the possibility of unfavorable new pre-clinical, clinical or safety data and further analyses of existing pre-clinical, clinical or safety data; risks associated with interim data, including the risk that final results from the Phase 3 trials for RSVpreF could differ from the interim data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; the ability to produce comparable clinical or other results for ABRYSVO (RSVpreF), Pfizer’s ATM-AVI candidate, PAXLOVID or any of Pfizer’s other infectious disease products or product candidates, including efficacy, safety and tolerability pro to date, in additional analyses of the Phase 3 trial and additional studies, in real world data studies or in larger, more diverse populations following commercialization; the ability of ABRYSVO (RSVpreF) and PAXLOVID to be effective against emerging virus variants; the risk that the use of ABRYSVO (RSVpreF), Pfizer’s ATM-AVI candidate, PAXLOVID or any of Pfizer’s other infectious disease products or product candidates will lead to new information about efficacy, safety, or other developments, including the risk of additional adverse reactions, some of which may be serious; the risk that pre-clinical and clinical trial data are subject to differing interpretations and assessments, including during the peer review/publication process, in the scientific community generally, and by regulatory authorities; whether and when additional data from ABRYSVO (RSVpreF), Pfizer’s ATM-AVI candidate, PAXLOVID or any of Pfizer’s other infectious disease products or product candidates will be published in scientific journal publications and, if so, when and with what modifications and interpretations; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when drug applications or submissions to request emergency use or conditional marketing authorizations for any potential indications for ABRYSVO (RSVpreF), Pfizer’s ATM-AVI candidate, PAXLOVID or any of Pfizer’s other infectious disease products or product candidates may be filed in any jurisdictions and if obtained, whether or when such emergency use authorization or licenses will expire or terminate; whether and when any applications that may be pending or filed for ABRYSVO (RSVpreF), Pfizer’s ATM-AVI candidate, PAXLOVID or any of Pfizer’s other infectious disease products or product candidates (including any requested amendments to the emergency use or conditional marketing authorizations) may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether ABRYSVO (RSVpreF), Pfizer’s ATM-AVI candidate, PAXLOVID or any of Pfizer’s other infectious disease products or product candidates will be commercially successful; intellectual property and other litigation; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of ABRYSVO (RSVpreF), Pfizer’s ATM-AVI candidate, PAXLOVID or any of Pfizer’s other infectious disease products or product candidates, including the authorization or approval of products or therapies developed by other companies; the risk that demand for any of our products may be reduced, no longer exist or not meet expectations, which may lead to excess inventory on-hand and/or in the channel, inventory write-offs or reduced revenues; challenges related to and uncertainties regarding the timing of a transition to the commercial market for any of our products; uncertainties related to the public’s adherence to vaccines and boosters; risks related to our ability to achieve our revenue forecasts for any of Pfizer’s infectious disease products or product candidates; the risk that other companies may produce superior or competitive products; risks related to the availability of raw materials to manufacture or test Pfizer’s infectious disease products or product candidates; challenges related to our vaccine’s formulation, dosing schedule and attendant storage, distribution and administration requirements, including risks related to storage and handling after delivery by Pfizer; the risk that we may not be able to successfully develop other vaccine formulations, booster doses or potential future annual boosters or re-vaccinations or new variant-based or next generation vaccines or potential combination respiratory vaccines; whether and when additional supply or purchase agreements will be reached or existing agreements will be completed or renegotiated; uncertainties regarding the ability to obtain recommendations from vaccine advisory or technical committees and other public health authorities regarding ABRYSVO (RSVpreF) or any of Pfizer’s other infectious disease products or product candidates and uncertainties regarding the commercial impact of any such recommendations; pricing and access challenges; challenges related to public confidence in, or awareness of ABRYSVO (RSVpreF), Pfizer’s ATM-AVI candidate, PAXLOVID or any of Pfizer’s other infectious disease products or product candidates; uncertainties regarding the impact of COVID-19 on our business, operations and financial results; and competitive developments. A further description of risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2022, and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors” and “Forward-Looking Information and Factors That May Affect Future Results”, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at and .

2023年5月12日，FDA咨询委员会讨论了Sarepta的杜氏肌营养不良症（DMD）基因疗法Elevidys的审批，14名委员会成员在经过了一整天的讨论后，以8票赞成、6票反对的微弱优势，建议FDA批准这款针对DMD患者的基因疗法，为这个被FDA质疑的项目带来了一丝光明。最终，FDA顺应了咨询委员会的建议，加速批准了Elevidys，使其成为美国历史上首个针对杜氏肌营养不良症的一次性疗法。那么，FDA的咨询委员会在FDA审批药物的监管过程中，扮演着怎样的角色？他们的建议对于FDA的决定产生多大的影响？在什么样的情况下，FDA会召集咨询委员会会议？药渡今天带你一探究竟！PART.01何为FDA咨询委员会？FDA咨询委员会（Advisory committees，简称Adcomm）是FDA负责牵头组建的，就人类和兽药、疫苗及其他生物制品、医疗器械和食品相关的问题，向FDA提供外部专家独立建议的临时机构。一般来说，FDA咨询委员会包括一名主席、几名成员，以及消费者代表、行业界代表各一名，有时还有一名患者代表。根据需要，可以为个别Adcomm会议增加具有特殊知识的其他专家。尽管委员会向FDA提供建议，但最终决定仍由FDA做出。也就是说，FDA可以做出与Adcomm相反的决定。“内事不决问张昭，外事不决问周瑜”，FDA在很多类似Sarepta的基因疗法这样的审批项目拿不定主意时，就会依靠Adcomm来帮助他们根据现有科学做出正确的决定。咨询委员会会议可以涉及广泛的科学讨论，例如开发疫苗预防COVID-19。PART.02专业知识、透明度和外部观点FDA有49个技术及科学咨询委员会和小组，其中包括科学专家和公众成员。Adcomm成员是根据他们的专业知识来选择的，以满足每个特定Adcomm的职能和主题领域的需求。Adcomm成员具有广泛的背景分布，例如医师研究人员、统计学家、工程师、化学家和生物学家等。此外，每个委员会通常包括一名消费者代表和一名行业代表。并根据会议主题，选择一名患者代表，为特定疾病或医疗产品提供患者或护理人员的经验。FDA启用Adcomm的原因及意义：从FDA外部的专家获得建议；努力建设一个公开透明的政府机构；鼓励患者、医疗保健提供者和其他感兴趣的人，在公开听证会期间提交评论来分享他们的观点。PART.03委员会提供科学、医学建议FDA咨询委员会的主要作用是在FDA评估产品时提供独立的专家建议，并帮助FDA根据现有科学做出合理的决定。委员会审查和评估项目，以及FDA监管的任何产品的安全性、有效性和相关数据。人类和兽药、生物制品、医疗器械、烟草和食品有关的问题都包含在其中。例如是否批准药物、设备或更新现有产品的标签。除了具体的项目讨论之外，委员会也讨论一般政策问题。Adcomm会议的过程是透明和独立的，并向公众开放。公众有机会向委员会发表言论，并分享他们的见解。在通常情况下，Adcomm会议会进行网络直播，以确保透明的讨论，并帮助公众了解FDA对产品和研究数据的期望。FDA通常在Adcomm会议召开前两个工作日向公众提供咨询委员会会议材料，这些材料包括来自FDA的议程和背景信息。虽然Adcomm的讨论对于FDA的决策非常关键，但更关键的是，Adcomm的建议对于FDA不具约束力。虚心纳谏，并不意味着言听计从。FDA通常会遵循Adcomm的建议，但不受法律约束。最终的决策权仍然在FDA手中牢牢把握。根据Milbank Quarterly上的一项统计研究发现，FDA对于大约80%的Adcomm的建议给予了支持，而对于20%则做出南辕北辙的决定。该研究调查了2008年至2015年间举行的376次AdComm会议，涉及298种产品或产品类别。该研究发现，FDA不遵循AdComm建议的主要因素，是AdComm成员本身就未能通过投票达成共识。那些票数微弱多数的观点，很有可能被FDA最终否决。换句话说，Elevidys的支持反对票数8:6，只是一个单次投票结果，可能并不具有统计学显著性。在这种情况下，FDA更有可能做出相对保守的决定，而不是微弱优势方代表的更激进冒险的观点。PART.04为什么FDA只将某些药物引向Adcomm？在选择咨询Adcomm时，FDA是否有明确透明的标准来选择产品项目？这是目前争议比较大的一个话题。而且对于FDA为什么有时会批准Adcomm反对的药物，质疑声音也很大。例如，在2021年FDA对aducanumab进行了有争议的批准，但批准之前，Adcomm给出了压倒性的反对票。FDA如果能够开诚布公地列举出Adcomm投票项目的标准，会帮助制药行业更清楚地洞察他们的产品是否会在批准之前接受Adcomm的审查，以及准备听证会可能提出的问题。值得注意的是，一份研究表明，2011年至2021年间，FDA将批准药物提交Adcomm审查的使用率从每年59%减少到6%，这是一个非常值得审视的信号，这更加引发了人们对FDA对于Adcomm选择标准的疑问。研究对象包含482种新药和生物制品，其中114种药物（24%）在FDA批准之前由Adcomm审查。这个平均数由2011年的59%一路飞流直下地跌到2021年6%的水平。研究人员非常关注那些被Adcomm反对，但却被FDA批准的药物。他们表示，这类产品每年的中位数是1个。在接受Adcomm审查的这114款药物中，Adcomm对其中的104种药物（91%）投了赞成票，对10种药物（9%）投了反对票。那些遭到Adcomm反对，但仍然被FDA“一意孤行”地通过的药物，包括2011年的罗氟司特（roflumilast）、2012年的florbetapir和omacetaxine mepesuccinate、2014 年的奥拉帕尼（olaparib）、2015年的帕比司他（panobinostat）和氟班色林（flibanserin）、2016年的eteplirsen、2019年的selinexor、2020年的oliceridine 和2021年的aducanumab。他们就像在足球比赛中起初被裁判宣判无效的进球，在经过VAR（视频助理裁判）审查后又失而复得被判有效的进球一样。对于反对票，Adcomm成员在讨论中最常见的顾虑是安全性和有效性（6 例）以及替代终点缺乏临床获益的证据（3例）。PART.05关于Adcomm的争论世异时移，FDA如今也在探索如何改革其咨询委员会，以确保这个组织能够充分发挥它们的作用。从2010年到2021年，FDA举行了409次与人类药物相关的会议，但这些会议召开的频率在逐渐降低：从2012年的50次减少到2020年和2021年的18次。Adcomm会议频率的降低，主要表现在初步批准投票的委员会会议上，从2012年的26次降低到 2021年的8次。有人的地方就存在争议。Adcomm的争议性在FDA加速批准Biogen的阿尔茨海默病药物Aduhelm（aducanumab）的事件上达到顶峰。当时Adcomm的几名成员一致投票反对Aduhelm的批准，但FDA最终并没有采纳投票决定，导致该小组成员辞职，以抗议FDA的一意孤行。Aduhelm的后来者Leqembi（lecanemab, Eisai和Biogen的阿尔茨海默病药物）在7月7日得到了FDA的全面批准，虽然Adcomm委员一致通过，但也并非没有争议，因为委员会中仅有6名成员。FDA在Leqembi的咨询委员会会议召开的最后一刻，增加了宾夕法尼亚州Abington Neurological Associates的神经学家David Weisman。他在过去的五年里，从Eisai和Biogen那里获得了数万美元，并直言不讳地表示支持Aduhelm。Weisman的入选使得Adcomm的公正性面临质疑。有人提出了建立生物伦理委员会，来审查Adcomm委员的资格，也有人甚至提出了废除Adcomm的建议。截至今年6月底，Adcomm在已经召开的19次Adcomm会议中，他们做出了18次批准药品的决定。FDA认为保留Adcomm投票流程是有益的，因为投票结果对于FDA来说并不具备强制性的效果，而是为他们做出最终决定起到一个参考作用。尤其是当Adcomm委员会对某一申请一致性的投票结果，与FDA的决定产生分歧之时，后者需要十分审慎地对待。如果FDA与Adcomm态度一致，FDA最终的决定也显得有理有据，增加了人们对监管结果的信任程度；如果FDA与Adcomm的态度出现分歧，也应该令公众了解实际情况，例如Aduhelm的评审。所以FDA认为，Adcomm策略是一种双赢的部署。虽然咨询委员会的建议不会也不应该决定监管决策的结果，但生物制药行业大多认为咨询委员会成员，包括参与Adcomm会议的每个无投票权的行业代表，都是在严格的人类药物审查过程的重要组成部分。因此保留这个组织，尽管存在争议，仍然是必要的。*附表：FDA近日主要Adcomm会议参考资料：1. McKenzie, H. 9 Highly Anticipated FDA Decisions in the Second Half of 2023. Biospace. 29. 06. 2023.1. Advisory Committees Give FDA Critical Advice and the Public a Voice. FDA.gov. 21. 09. 2021.2. Craven, J. FDA's use of adcomms prior to drug approval decreased over the last decade. Regulatory Focus. 03. 05. 2022.3. Dearment, A. FDA often goes against advisory committee recommendations when votes are divided, study finds. MedCityNews. 16. 06. 2019.4. Recent And Upcoming FDA Advisory Committee Meetings. Pink Sheet. 07. 07. 2023.5. Brennan, Z. No more FDA adcomm votes? Reforms brew as new research highlights decline in meetings. Endpoints News. 07. 07. 2023.封面图来源：网络两大巨头打响RSV「首秀」争夺战，GSK直接釜底抽薪：辉瑞你就别卖了...K药或成“最短寿”药王？凯风创投王立铭：first-in-class或为伪命题，中国创新药面临时代主题切换PHDI 2023 倒计时1个月丨30+业内大咖云集制药产业数智化峰会点击这里，与药时代一起快乐学习！