MIPLYFFA, the first FDA-approved treatment for Niemann-Pick disease type C, is available at Zevra's specialty pharmacy for dispense
AmplifyAssist, Zevra's comprehensive patient support program, in place to address access barriers
CELEBRATION, Fla., Nov. 21, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage rare disease therapeutics company, today announced that MIPLYFFA (MY-PLY-FAH) (arimoclomol), the first treatment approved by the U.S. Food and Drug Administration (FDA) for the treatment of Nieman Pick disease type C (NPC), is now commercially available for dispense. MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients 2 years of age and older.
"Early adoption of MIPLYFFA has exceeded our expectations, and we are pleased to report that product is now available for shipment to NPC patients. This is in line with the shorter timeframe of our guidance of eight to 12 weeks post-approval," said Josh Schafer, Chief Commercial Officer and Executive Vice President of Business Development, Zevra Therapeutics. "We continue to receive prescription enrollments through our patient services program, AmplifyAssist, and are supporting patients as they navigate the benefits verification process to obtain MIPLYFFA."
AmplifyAssist is Zevra's comprehensive patient support program designed to support the individual needs of eligible patients and those who care for them. Available resources include personalized insurance coverage education and support, copay and alternate funding identification assistance for eligible patients' product needs, disease state information and therapy management counseling, and ongoing interactions to address barriers while facilitating timely prescription refills. Information about the program is available at MIPLYFFA.com or via telephone. The AmplifyAssist team can be reached toll-free at (888) 668-4198 from 8 a.m. CT to 6 p.m. CT Monday through Friday.
Patients and caregivers should speak with their physician to get started with MIPLYFFA. Physicians can fill out an enrollment form at MIPLYFFA.com, order the prescription, and when approved, the medication will be mailed to their patient's home.
Healthcare providers and patients/caregivers should refer to the Full Prescribing Information and Instructions for Use for information on the proper administration of MIPLYFFA.
AmplifyAssist — Comprehensive Support for Patients
Zevra is committed to assisting those whose lives are affected by NPC to overcome the barriers and challenges that may impact their treatment journey. AmplifyAssist is Zevra's comprehensive patient support program. The mission of the program is to support the individual needs of eligible patients and those who care for them. Available resources include personalized insurance coverage education and support, copay and alternate funding identification assistance for eligible patients' product needs, disease state information and therapy management counseling, and ongoing interactions to address barriers while facilitating timely prescription refills. Information about the program is available at MIPLYFFA.com or via telephone. The AmplifyAssist team can be reached toll-free at (888) 668-4198 from 8 a.m. CT to 6 p.m. CT Monday through Friday. Healthcare providers who want to submit prescriptions can visit MIPLYFFA.com to complete the prescription enrollment form that initiates the process for accessing the treatment.
About MIPLYFFA (arimoclomol)
MIPLYFFA (arimoclomol) increases the activation of the transcription factors EB (TFEB) and E3 (TFE3) resulting in the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes. MIPLYFFA has also been shown to reduce unesterified cholesterol in the lysosomes of human NPC fibroblasts. The clinical significance of these findings is not fully understood. In the pivotal phase 3 trial, MIPLYFFA halted disease progression compared to placebo over the one-year duration of the trial when measured by the only validated disease progression measurement tool, the NPC Clinical Severity Scale. MIPLYFFA was granted Breakthrough Therapy designation, Rare Pediatric Disease designation, Orphan Drug designation, and Fast Track designation by the FDA for the treatment of NPC. MIPLYFFA was further granted Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC.
INDICATIONS AND USAGE
MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older.
IMPORTANT SAFETY INFORMATION
Hypersensitivity Reactions:
Hypersensitivity reactions such as urticaria and angioedema have been reported in patients treated with MIPLYFFA during Trial 1: two patients reported both urticaria and angioedema (6%) and one patient (3%) experienced urticaria alone within the first two months of treatment. Discontinue MIPLYFFA in patients who develop severe hypersensitivity reactions. If a mild or moderate hypersensitivity reaction occurs, stop MIPLYFFA and treat promptly. Monitor the patient until signs and symptoms resolve.
Embryofetal Toxicity:
MIPLYFFA may cause embryofetal harm when administered during pregnancy based on findings from animal reproduction studies. Advise pregnant females of the potential risk to the fetus and consider pregnancy planning and prevention for females of reproductive potential.
Increased Creatinine without Affecting Glomerular Function:
Across clinical trials of MIPLYFFA, mean increases in serum creatinine of 10% to 20% compared to baseline were reported. These increases occurred mostly in the first month of MIPLYFFA treatment and were not associated with changes in glomerular function.
During MIPLYFFA treatment, use alternative measures that are not based on creatinine to assess renal function. Increases in creatinine reversed upon MIPLYFFA discontinuation.
The most common adverse reactions in Trial 1 (≥15%) in MIPLYFFA-treated patients who also received miglustat were upper respiratory tract infection, diarrhea, and decreased weight.
Three (6%) of the MIPLYFFA-treated patients had the following adverse reactions that led to withdrawal from Trial 1: increased serum creatinine (one patient), and progressive urticaria and angioedema (two patients). Serious adverse reactions reported in MIPLYFFA-treated patients were hypersensitivity reactions including urticaria and angioedema.
To report SUSPECTED ADVERSE REACTIONS, contact Zevra Therapeutics, Inc. at toll-free phone 1-844-600-2237 or FDA at 1‐800-FDA-1088 or .
Drug Interaction(s):
Arimoclomol is an inhibitor of the organic cationic transporter 2 (OCT2) transporter and may increase the exposure of drugs that are OCT2 substrates. When MIPLYFFA is used concomitantly with OCT2 substrates, monitor for adverse reactions and reduce the dosage of the OCT2 substrate.
Use in Females and Males of Reproductive Potential:
Based on animal findings, MIPLYFFA may impair fertility and may increase post-implantation loss and reduce maternal, placental, and fetal weights.
Renal Impairment:
The recommended dosage of MIPLYFFA, in combination with miglustat, in patients with an eGFR ≥15 mL/minute to <50 mL/minute is lower than the recommended dosage (less frequent dosing) in patients with normal renal function.
MIPLYFFA capsules for oral use are available in the following strengths: 47 mg, 62 mg, 93 mg, and 124 mg.
About Niemann-Pick Disease Type C (NPC)
Niemann-Pick disease type C (NPC) is an ultra-rare, progressive, and neurodegenerative lysosomal storage disorder characterized by an inability of the body to transport cholesterol and other lipids within the cell, leading to an accumulation of these substances in various cell types, including neurons. The disease is caused by mutations in the NPC1 or NPC2 genes, which are responsible for making the NPC1 and NPC2 lysosomal proteins. Both children and adults can be affected by NPC with varying clinical presentations. Those living with NPC can lose independence due to physical and cognitive limitations, with key neurological impairments presenting in speech, cognition, swallowing, ambulation, and fine motor skills. Disease diagnosis can often take years, with disease progression being irreversible and often leading to early mortality.
About Zevra Therapeutics, Inc.
Zevra Therapeutics, Inc. is a commercial-stage rare disease company combining science, data, and patient needs to create transformational therapies for diseases with limited or no treatment options. Our mission is to bring life-changing therapeutics to people living with rare diseases. With unique, data-driven development and commercialization strategies, the Company is overcoming complex drug development challenges to make new therapies available to the rare disease community.
Expanded access programs are made available by Zevra Therapeutics, Inc. and its affiliates and are subject to the Company's Expanded Access Program (EAP) policy, as published on its website. Participation in these programs is subject to the laws and regulations of each jurisdiction under which each respective program is operated. Eligibility for participation in any such program is at the treating physician's discretion.
For more information, please visit or follow us on X (formerly Twitter) and LinkedIn.
Cautionary Note Concerning Forward-Looking Statements
This press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that do not relate solely to historical or current facts, including without limitation statements regarding the potential benefits of any of our products or product candidates for any specific disease or at any dosage; our strategic and product development objectives; prescription enrollments; our ability to support patients as they navigate the benefits verification process to obtain MIPLYFFA; and availability of and access to MIPLYFFA. Forward-looking statements are based on information currently available to Zevra and its current plans or expectations. They are subject to several known and unknown uncertainties, risks, and other important factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. These and other important factors are described in detail in the "Risk Factors" section of Zevra's Annual Report on Form 10-K for the year ended December 31, 2023, Zevra's quarterly report for the three and nine months ended September 30, 2024, and Zevra's other filings with the Securities and Exchange Commission. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we cannot assure that such expectations will prove correct. These forward-looking statements should not be relied upon as representing our views as of any date after the date of this press release.
_____________________________________________
Zevra Contact
Nichol Ochsner
+1 (732) 754-2545
nochsner@zevra.com
Russo Partners Contacts
David Schull
+1 (858) 717-2310
david.schull@russopartnersllc.com
Ignacio Guerrero-Ros, Ph.D.
+1 (646) 942-5604
ignacio.guerrero-ros@russopartnersllc.com
MIPLYFFA，第一个获得FDA批准用于尼曼-皮克氏病C型的治疗的药物，在Zevra的专科药房可供配药
AmplifyAssist，Zevra的综合患者支持计划旨在解决获取障碍
2024年11月21日，佛罗里达州庆祝（GLOBE NEWSWIRE）--Zevra Therapeutics, Inc.（NasdaqGS: ZVRA）（Zevra或公司），一家商业阶段罕见病疗法公司，今天宣布，MIPLYFFA（MY-PLY-FAH）（arimoclomol），第一款获得美国食品药品监督管理局（FDA）批准用于治疗尼曼-皮克氏病C型（NPC）的治疗药物，现已可供商业配药。MIPLYFFA适用于与米格鲁斯他共同用于治疗2岁及以上成人和儿童NPC的神经表现。
“MIPLYFFA的早期采用超出了我们的预期，我们很高兴地宣布产品现在可供配送给NPC患者。这符合我们八到十二周获批后的指导时间表，”Zevra Therapeutics的首席商业官兼业务发展执行副总裁Josh Schafer说。“我们继续通过我们的患者服务计划AmplifyAssist接收处方登记，并支持患者在获取MIPLYFFA时进行福利核实流程。”
AmplifyAssist是Zevra的综合患者支持计划，旨在支持符合条件的患者及其照顾者的个体需求。可用资源包括个性化保险覆盖教育和支持，报销和替代资金识别协助符合条件患者的产品需要，疾病状态信息和治疗管理咨询，以及持续互动，以解决障碍，同时促进及时处方补充。有关该计划的信息，请访问MIPLYFFA.com或致电。从周一到周五早上8点到下午6点，可以通过免费电话(888) 668-4198联系AmplifyAssist团队。
患者和照顾者应与其医生交谈，开始使用MIPLYFFA。医生可以在MIPLYFFA.com填写入学表格，下订单处方，经批准后，药物将邮寄到他们患者的家中。
医疗保健提供者和患者/照顾者应参考MIPLYFFA的完整处方信息和使用说明，了解MIPLYFFA的正确使用方法。
AmplifyAssist — 患者的全面支持
Zevra致力于帮助那些生活受到NPC影响的人克服可能影响他们治疗旅程的障碍和挑战。AmplifyAssist是Zevra的综合患者支持计划。该计划的使命是支持符合条件的患者及照料他们的人的个人需求。可用资源包括个性化保险覆盖教育和支持、适用患者产品需求的copay和备用资金识别援助、疾病状态信息和治疗管理咨询，以及持续互动以解决障碍并促进及时处方补充。有关该计划的信息可在MIPLYFFA.com或通过电话获取。可通过免费电话(888) 668-4198与AmplifyAssist团队联系，服务时间为周一至周五上午8点至下午6点。希望提交处方的医疗保健提供者可以访问MIPLYFFA.com填写处方注册表格，启动访问治疗流程。
关于MIPLYFFA（arimoclomol）
MIPLYFFA（阿利莫克雷唑）增加了转录因子Eb（TFEB）和E3（TFE3）的激活，导致协调溶酶体表达和调控（CLEAR）基因的上调。MIPLYFFA还显示能够降低人类NPC成纤维细胞溶酶体中的游离胆固醇。这些发现的临床意义尚未完全理解。在关键的3期试验中，MIPLYFFA与安慰剂相比在试验持续一年期间能够阻止疾病进展，这是通过唯一验证的疾病进展测量工具NPC临床严重度评分进行衡量的。MIPLYFFA获得了FDA授予的突破性治疗药物、罕见儿科疾病、孤儿药物和快速通道治疗指定，用于治疗NPC。MIPLYFFA还获得了欧洲药品管理局（EMA）授予的NPC治疗孤儿药品指定。
LINZESS（linaclotide）适用于治疗成人肠易激综合征伴便秘（IBS-C）和慢性特发性便秘（CIC），以及6至17岁儿童和青少年的便秘功能障碍（FC）。不知道LINZESS在年龄小于6岁的FC儿童或IBS-C小于18岁的患者中是否安全和有效。
MIPLYFFA适用于与米格鲁斯他联用，治疗2岁及以上的成人和儿童患者的尼曼-皮克病C型（NPC）神经表现。
重要安全信息
过敏反应：
在试验1期间，有关MIPLYFFA治疗的患者报告了过敏反应，如荨麻疹和血管性水肿：两名患者报告出现荨麻疹和血管性水肿（6%），一名患者（3%）在治疗的前两个月内仅出现荨麻疹。对于出现严重过敏反应的患者，请停止使用MIPLYFFA。如果出现轻度或中度过敏反应，请停止使用MIPLYFFA并及时处理。监测患者直至症状消失。
胚胎毒性：
MIPLYFFA可能会导致胚胎和胎儿受损，根据动物繁殖研究结果。建议怀孕的女性注意胎儿潜在风险，并考虑对生育潜力的女性进行妊娠规划和预防。
肌酐增高但不影响肾小球功能：
在MIPLYFFA的临床试验中，与基线相比，血清肌酐平均增加了10%到20%。这些增加主要发生在MIPLYFFA治疗的第一个月，与肾小球功能的变化无关。
MIPLYFFA治疗期间，请使用不基于肌酐的替代措施评估肾功能。停用MIPLYFFA后，肌酐水平会恢复正常。
在接受MIPLYFFA治疗并使用米格鲁斯他的患者中，试验1中（≥15%）最常见的不良反应是上呼吸道感染、腹泻和体重减轻。
MIPLYFFA治疗的患者中有3人（6％）出现了以下导致退出试验1的不良反应：血清肌酐增高（1例患者），进行性荨麻疹和血管性水肿（2例患者）。MIPLYFFA治疗的患者报告的严重不良反应包括荨麻疹和血管性水肿的过敏反应。
如有可疑不良反应，请联系Zevra Therapeutics, Inc.免费电话1-844-600-2237或致电FDA 1‐800-FDA-1088或。
药物相互作用：
Arimoclomol是有机阳离子转运蛋白2(OCT2)的抑制剂，可能增加是OCT2底物的药物的曝光量。当MIPLYFFA与OCT2底物同时使用时，请监测不良反应并减少OCT2底物的剂量。
用于生育潜力的女性和男性中。
根据动物实验结果，MIPLYFFA可能会影响生育能力，增加植入后流失，并减少母体、胎盘和胎儿的重量。
肾功能损伤：
MIPLYFFA的建议剂量与米格鲁斯他联用，在eGFR≥15 mL/minute至<50 mL/minute的患者中，比正常肾功能患者的建议剂量更低（服药频率更低）。
MIPLYFFA口服胶囊有以下几种规格：47毫克，62毫克，93毫克和124毫克。
关于尼曼-匹克病C型（NPC）
尼曼-匹克病C型（NPC）是一种极其罕见、进行性和神经退行性溶酶体贮积症，其特点是身体无法在细胞内输送胆固醇和其他脂质，导致这些物质在包括神经元在内的各种细胞类型中积累。该疾病由NPC1或NPC2基因突变引起，这些基因负责产生NPC1和NPC2溶酶体蛋白。儿童和成人均可能受到NPC的影响，临床表现不同。NPC患者可能由于身体和认知功能的限制而失去独立性，关键神经系统损伤表现为言语、认知、吞咽、行走和精细运动技能方面的障碍。该疾病的诊断往往需要多年时间，疾病进展不可逆转，常导致早期死亡。
关于Zevra Therapeutics, Inc.
Zevra Therapeutics, Inc.是一家商业化阶段的罕见病公司，结合科学、数据和患者需求，为那些没有或有限治疗选择的疾病开发转变性疗法。我们的使命是为罕见病患者带来改变生活的疗法。通过独特的数据驱动的研发和商业化策略，公司正在克服复杂的药物开发挑战，使新疗法能够服务罕见病社区。
Zevra Therapeutics公司及其关联公司提供扩展获取计划，并遵守公司的扩展获取计划（EAP）政策，该政策已在其网站上发布。参与这些计划需遵守各个辖区的法律法规。参与任何此类计划的资格由治疗医师自行决定。
欲了解更多信息，请访问 或关注我们X（前Twitter）和LinkedIn。
关于前瞻性声明的注意事项
本新闻稿可能包含根据1995年《私人证券诉讼改革法案》的前瞻性声明。前瞻性声明包括所有不仅涉及历史事实或当前事实的声明，还包括关于我们任何产品或产品候选者对于任何特定疾病或任何剂量的潜在好处的声明；我们的战略和产品开发目标；处方登记；我们支持患者在获得MIPLYFFA的利益验证过程中的能力；以及MIPLYFFA的可用性和获取途径。前瞻性声明基于Zevra目前可获得的信息以及其当前计划或期望。它们受到几个已知和未知的不确定因素、风险和其他重要因素的影响，可能导致我们的实际结果、表现或成就与前瞻性声明所表达或暗示的任何未来结果、表现或成就有实质不同。这些以及其他重要因素在Zevra于2023年12月31日结束的年度10-k表格上的“风险因素”部分，Zevra于2024年9月30日结束的三个月和九个月的季度报告以及Zevra向证券交易委员会提交的其他文件中有详细描述。尽管我们可能会选择在将来的某个时间更新此类前瞻性声明，但除非法律要求，我们拒绝这样做，即使随后发生的事件导致我们的看法发生变化。虽然我们相信此类前瞻性声明所体现的期望是合理的，但我们不能保证这些期望将被证明是正确的。这些前瞻性声明不应被依赖为代表我们在本新闻稿发布日期后的任何日期的观点。
_____________________________________________
Zevra联系方式
Nichol Ochsner
+1 (732) 754-2545
nochsner@zevra.com
Russo Partners 联系方式
David Schull
+1 (858) 717-2310
david.schull@russopartnersllc.com
Ignacio Guerrero-Ros博士。
+1 (646) 942-5604
ignacio.guerrero-ros@russopartnersllc.com
