Hypokalemic Periodic Paralysis

看点• 8月CDE新增一致性评价受理号54个（37个品种、35家企业）。• 8月有199个受理号（含视同通过）涉及117个品种过评。2023年8月，一致性评价申报数量在7月回暖后再次降低，直逼2023年全年最低，仅54个受理号；但在一致性过评方面，8月通过/视同通过一致性评价受理号数量明显回升，共199个受理号过评（一致性评价补充申请受理号82个，仿制药视同通过受理号117个）。（文末附2023年8月申报与过评一致性评价详情表）PS：药智网公众号【ID:yaozh008】后台回复【8月一致性评价】，即可获取本文表格excel格式文档图1 2023年01月-2023年08月申报/通过趋势01过评详情2023年8月，有82个一致性评价补充申请受理号通过一致性评价，涉及48个企业的49个品种；还有117个新注册分类仿制药上市申请受理号视同通过一致性评价，较7月过评数量有明显提升。由于部分新批准药品还没能收集到详细信息等原因，导致一致性评价数据库中的数据和注册数据可能会存在差异，现在将整理出的2023年8月首家过评的11个药品信息放在文末表1中，供参考。从过评企业而言，8月共有199个受理号过评，涉及120家企业的117个品种，其中上海禾丰制药有限公司、山东鲁抗医药股份有限公司和成都青山利康药业股份有限公司均有3个品种过评，并列企业通过品种数排名第一，其余企业均为2个或1个品种过评。企业通过品种数详情见下图。图2 2023年08月企业通过品种数TOP10上海禾丰制药有限公司是中国优质高效经典急救普药研发和生产基地。公司主要生产小容量注射液、固体制剂、口服液三大剂型，包括心血管、神经系统、抗生素、维他命、消化系统、抗疟类等九大类产品。公司申报一致性评价的剂型均为注射剂，暂无其他制剂一致性评价相关信息。目前已有8个品种通过/视同通过一致性评价，盐酸多巴胺注射液为首家过评企业，8月通过的3个品种均为一致性评价补充申请过评，分别为：缩宫素注射液、盐酸去氧肾上腺素注射液和重酒石酸间羟胺注射液；公司还有3个注射剂品种还在审评审批过程中，暂无结论。山东鲁抗医药股份有限公司主要生产经营人用抗生素、半合成抗生素、兽用农用抗生素、生物技术药品以及抗生素相关制剂、医药中间体、药用树脂、葡萄糖等产品。目前已有21个品种通过一致性评价补充申请，其中3个品种为首家过评，7个品种以新注册分类仿制药视同通过一致性评价，还有6个品种在审评过程中；其中8月过评的3个品种分别为：头孢克肟片、头孢克洛颗粒、枸橼酸托法替尼片。成都青山利康药业股份有限公司是集药品、医疗器械、化妆品研发、生产、销售于一体的高新技术企业。公司共有4个品种通过/视同通过一致性评价，其中氨基酸(15)腹膜透析液为国内首家以新注册分类仿制药视同通过一致性评价，8月过评的低钙腹膜透析液(乳酸盐-G1.5%)、低钙腹膜透析液(乳酸盐-G2.5%)、低钙腹膜透析液(乳酸盐-G4.25%)均为一致性评价补充申请过评。从品种看，2023年8月共有117个品种过评，品种通过受理号数top10中，沙库巴曲缬沙坦钠片以3家企业8个受理号过评位居第一，其次为注射用拉氧头孢钠5个受理号2个厂家过评位列第二。详情见下图。图3 2023年8月品种通过受理号数TOP10沙库巴曲缬沙坦钠片属于医保乙类处方药，是一种用于射血分数降低的慢性心力衰竭成人患者的降压药。目前仅有3家企业以新注册分类仿制药视同通过一致性评价，均为8月过评，分别为南京一心和医药科技有限公司、石药集团欧意药业有限公司和正大天晴药业集团股份有限公司；还有17家企业以提交新注册分类仿制药上市申请并受理，14家企业在一致性进度数据库中查询到有临床试验信息。注射用拉氧头孢钠属于医保乙类处方药，适应症为用于敏感菌引起的各种感染症，如败血症、脑膜炎、呼吸系统感染症(肺炎、支气管炎、支气管扩张症、肺化脓症、脓胸等)，消化系统感染症(胆道炎、胆囊炎等)，腹腔内感染症(肝脓疡、腹膜炎等)，泌尿系统及生殖系统感染症(肾孟肾炎、膀胱炎、尿道炎、淋病、副睾炎、子宫内感染、子宫附件炎、盆腔炎等)，皮肤及软组织感染、骨、关节感染及创伤感染。目前已有4家企业通过一致性评价，3家企业已新注册分类仿制药视同通过一致性评价，1家企业提交一致性评价补充申请，3家企业提交新注册分类仿制药上市申请。8月过评的两家企业均以一致性评价补充申请过评，分别为杭州森泽医药科技有限公司和海南灵康制药有限公司。0237个品种申报，注射剂近四分之三2023年8月份CDE新增一致性评价受理号54个,涉及35家企业的37个品种，其中注射剂申报占比超百分之七十，片剂过百分之二十。图4 2023年8月申报剂型详情申报品种而言，2023年8月，注射用头孢哌酮钠舒巴坦钠（2:1）有4个受理号申报位列第一，氟马西尼注射液、氯化钾注射液、注射用青霉素钠各有3个受理号申报，并列第二。详情见下图图5 2023年8月品种申报受理号数TOP10注射用头孢哌酮钠舒巴坦钠（2:1）用于治疗由敏感细菌所引起的感染，适应症包括呼吸道感染,尿路感染,腹膜炎,胆囊炎,胆管炎,败血症,脑膜炎,皮肤及软组织感染,骨关节炎,盆腔炎,子宫内膜炎,淋病,呼吸内科,肾病内科,普外科,消化内科,肝胆外科,传染病科,神经内科,皮肤科,骨科,妇产科,性病科。注射用头孢哌酮钠舒巴坦钠（2:1）目前暂无通过/视同通过一致性评价品种，但已有3家企业提交一致性评价补充申请或新注册分类仿制药上市申请。氟马西尼注射液适应症为用于逆转苯二氮䓬类药物所致的中枢镇静作用：1．终止用苯二氮䓬类药物诱导及维持的全身麻醉。2．作为苯二氮䓬类药物过量时中枢作用的特效逆转剂。3．用于鉴别诊断苯二氮䓬类、其它药物或脑损伤所致的不明原因的昏迷。目前已有4家企业通过一致性评价补充申请，1家企业视同通过一致性评价。宜昌人福药业有限责任公司为首家过评企业，目前该品种还有4家企业已提交一致性评价补充申请或新注册分类仿制药上市申请。氯化钾注射液适应症为1.治疗各种原因引起的低钾血症，如进食不足、呕吐、严重腹泻、应用排钾性利尿药、低钾性家族周期性麻痹、长期应用糖皮质激素和补充高渗葡萄糖后引起的低钾血症等。2.预防低钾血症，当患者存在失钾情况，尤其是如果发生低钾血症对患者危害较大时（如使用洋地黄类药物的患者），需预防性补充钾盐，如进食很少、严重或慢性腹泻、长期服用肾上腺皮质激素、失钾性肾病、Bartter综合征等。3.洋地黄中毒引起频发性、多源性早搏或快速心律失常。目前该产品虽只有2家企业通过一致性评价，但有22家企业已提交一致性评价补充申请或新注册分类仿制药上市申请。注射用青霉素钠适应症为青霉素适用于敏感细菌所致各种感染，如脓肿、菌血症、肺炎和心内膜炎等。其中青霉素为以下感染的首选药物：1．溶血性链球菌感染，如咽炎、扁桃体炎、猩红热、丹毒、蜂窝织炎和产褥热等。2．肺炎链球菌感染如肺炎、中耳炎、脑膜炎和菌血症等。3．不产青霉素酶葡萄球菌感染。4．炭疽。5．破伤风、气性坏疽等梭状芽孢杆菌感染。6．梅毒(包括先天性梅毒)。7．钩端螺旋体病。8．回归热。9．白喉。10．青霉素与氨基糖苷类药物联合用于治疗草绿色链球菌心内膜炎。青霉素亦可用于治疗：1．流行性脑脊髓膜炎。2．放线菌病。3．淋病。4．奋森咽峡炎。5．莱姆病。6．鼠咬热。7．李斯特菌感染。8．除脆弱拟杆菌以外的许多厌氧菌感染。风湿性心脏病或先天性心脏病患者进行口腔、牙科、胃肠道或泌尿生殖道手术和操作前，可用青霉素预防感染性心内膜炎发生。该产品有2家企业过评一致性，一致性评价补充申请或新注册分类仿制药上市申请的企业还有9家。从企业角度，2023年8月，所有企业都只申报了1个品种，其中海南广升誉制药有限公司提交4个一致性评价补充申请受理号位居榜首，西南药业股份有限公司提交3个一致性评价补充申请受理号位居第二。详情见下图。图6 2023年8月企业申报受理号数TOP10海南广升誉制药有限公司成立于2016年01月18日，经营范围包括药品生产、销售，药品委托生产、加工，医药技术开发、咨询。拥有21个品种的74个上市批文，目前已有1个品种过评一致性，2个品种以提交一致性评价补充申请，3个品种查询到临床试验相关信息。西南药业股份有限公司位于山水之城，美丽之地——重庆，主要生产片剂、水针剂、粉针剂、输液剂、冻干剂、酊水剂及麻醉品。公司已有13个品种通过/视同一致性评价，10个品种提交一致性评价补充申请/新注册分类仿制药上市申请，8月申报品种为注射用青霉素钠。附表1：2023年8月首家通过一致性评价详情表附表2：2023年8月通过（含视同通过）一致性评价（过评厂家数为2-10）详情表附表3：2023年8月申报一致性评价详情表数据截止至2023年8月31日数据来源：药智数据企业版——仿制药一致性评价分析系统声明：本内容为作者独立观点，不代表药智网立场。如需转载，请务必注明文章作者和来源。对本文有异议或投诉，请联系maxuelian@yaozh.com。责任编辑 | 八角转载开白 | 马老师 18996384680（同微信）商务合作 |  张武龙 13368443108（同微信） 阅读原文，是昨天最受欢迎的文章哦

Major players in the Andersen-Tawil syndrome industry leverage the growing global interest in personalized medicine for non-standardized treatments. Fact.MR, Rockville MD: Market research firm Fact.MR’s has published a report on the Andersen-Tawil syndrome market has estimated positive prospects of growth in the near future. The resumption of elective medical treatment procedures in 2021 as the covid-19 pandemic subsides, will support recovery of demand arising in the industry. Further, removal of restrictions on research activities associated with rare diseases, will generate major opportunities for Andersen-Tawil syndrome treatment options in the years ahead. According to the National Organization for Rare Disorders, the Andersen-Tawil syndrome affects male and female demographics equally. However, only around 100 cases overall have been reported in global medical literature. Diseases such as thyrotoxic periodic paralysis, hypokalemic periodic paralysis, and hyperkalemic period paralysis display similar symptoms. Consequently, accurate diagnosis is considered as major challenge among market players. Some of the more prominent diagnostic and treatment options include potassium supplements, molecular genetic testing, voluntary muscle contraction exercises, Holter monitoring and electrocardiograms. Owing to unique symptoms among individual patients, treatments are likely to remain non-standardized for the foreseeable future. “Increased government funding efforts aimed towards improving diagnostic procedures is expected to generate lucrative opportunities for the participants in the Andersen-Tawil syndrome market. Non-profit organizations such as Periodic Paralysis International, Muscular Dystrophy UK, Sudden Arrhythmia Death Syndromes Foundation, and Periodic Paralysis Association, are playing key roles in raising awareness positively influencing market developments,” comments a Fact.MR analyst. Request a report sample to gain detailed insights at Key Takeaways from Fact.MR’s Andersen-Tawil Syndrome Market Beta blocker treatments are projected to reflect faster growth on the back of treatment efficacy and relatively lower costs. Genetic tests will hold larger market share owing to higher levels of accuracy in diagnostic procedures. Online pharmacies are rapidly gaining traction, with higher levels of internet penetration and ease of product access to patients. The United States accounts for significant market share owing to high numbers of insurance payers and a vast private health sector. Economic growth and investments into healthcare infrastructure support growth in China and India. Andersen-Tawil Syndrome Market – Prominent Drivers Easy international access to data on the Andersen-Tawil syndrome in recent years is supporting associated research efforts. Growing government investments and incentives towards rare disease research supports growth in the market. Growing involvement of private and public organizations supporting treatment and raising awareness will positively influence future developments. Andersen-Tawil Syndrome Market– Key Restraints Low prevalence of the Andersen-Tawil syndrome and lack of awareness remains a key problem hurting market players. High risk of misdiagnosis and undiagnosed cases continue as prominent issues in the industry. High costs associated to rare disease research will hold back innovations and growth prospects. Discover more about the Andersen-Tawil syndrome market with figures data tables and table of contents. You can also find detailed market segmentation at Competitive Landscape Leading players in the Andersen-Tawil syndrome market include but are not limited to Mylan N.V., Teva Pharmaceuticals Ltd., Novartis AG, Zydus Pharmaceuticals Inc., Advanz Pharmaceuticals, and Sun Pharmaceutical Industries Ltd. Leading players in the industry are encouraging research & development efforts in addition to strategic mergers and acquisitions to widen the scope of applications and consolidating their position in the market. In April 2021, Teva Pharmaceuticals was acquired by Karo Pharma Aktiebolag with the aim of gaining the European OTC brand portfolios and drug research including rare disease solutions. Also, Sun Pharmaceutical Industries Ltd. announced the acquisition of 66% ownership interest in Trikaal Mediinfotech, and AWACS, through its subsidiary ABCD Technologies LLP. More Insights on the Andersen-Tawil syndrome Market In its latest report, Fact.MR offers unbiased analysis of the global Andersen-Tawil syndrome market. In order to understand the global market potential, its growth, and scope, the market is segmented on the basis of disease type (type 1 and type 2), drug class (carbonic anhydrate inhibitors, acetazolemaide, dichlorpenamide, antiarrhythmic drugs, and beta blockers), and distribution channel (hospital pharmacies, retail pharmacies, and online pharmacies) across seven regions (North America, Latin America, Europe, East Asia, South Asia, Oceania, and Middle East & Africa). Request For No-Obligation Table Of Content Explore Fact.MR’s Coverage on the Healthcare Domain Toxic Shock Syndrome Treatment Market: A recent study by Fact.MR on the toxic shock syndrome treatment market offers a 8-year forecast for 2018 to 2026. The study analyzes key trends that are currently determining the growth of the market. This report covers vital dynamics, including drivers, restraints, and opportunities for leading market players along with key stakeholders and emerging players. Angelman Syndrome Treatment Market: Fact.MR’s extensive coverage on the Angelman syndrome treatment market offers in-depth insights about the prominent growth dynamics that are likely to aid expansion of prospects in the near future. Data has been presented in the form of key segments across prominent geographies, along with important information concerning key manufacturers operating within the landscape. Goodpasture Syndrome Treatment Market: The global good pasture syndrome treatment market study published by Fact.MR offers an unbiased analysis of the prominent drivers, opportunities and trends expected to shape future expansion outlook. The report provides a comprehensive assessment on the prominent segments and geographies which are likely to benefit market players in coming years. About Fact.MR Market research and consulting agency with a difference! That’s why 80% of Fortune 1,000 companies trust us for making their most critical decisions. We have offices in US and Dublin, whereas our global headquarter is in Dubai. While our experienced consultants employ the latest technologies to extract hard-to-find insights, we believe our USP is the trust clients have on our expertise. Spanning a wide range – from automotive & industry 4.0 to healthcare & retail, our coverage is expansive, but we ensure even the most niche categories are analyzed. Reach out to us with your goals, and we’ll be an able research partner. Contact: US Sales Office: 11140 Rockville Pike Suite 400 Rockville, MD 20852 United States Tel: +1 (628) 251-1583 E: sales@factmr.com Follow Us: LinkedIn | Twitter Market Intelligence Services: Marketngage.com Source: Fact.MR

~ Submitted New Drug Application (NDA) for RECORLEV® (levoketoconazole) for the Treatment of Endogenous Cushing’s Syndrome to U.S. Food & Drug Administration ~ ~ KEVEYIS® (dichlorphenamide) Full-Year 2020 Revenue of $30.7 Million, a 41.5 Percent Increase over 2019 Revenue of $21.7 Million ~ ~ Full-Year 2021 KEVEYIS® (dichlorphenamide) Revenue Guidance of $34 Million to $36 Million ~ ~ Company Reports $87.5 Million in Cash on Hand; Expects to Fund Operations Into and Potentially Beyond the First Quarter of 2023 ~ ~ Strongbridge to Host Conference Call Today at 8:30 a.m. ET ~ DUBLIN, Ireland and TREVOSE, Pa., March 03, 2021 (GLOBE NEWSWIRE) -- Strongbridge Biopharma plc, (Nasdaq: SBBP), a global commercial-stage biopharmaceutical company focused on the development and commercialization of therapies for rare diseases with significant unmet needs, today reported financial results for the fourth quarter and full year ended December 31, 2020. “With the successes of 2020 as a backdrop, we have begun 2021 with great momentum and optimism. Based upon the two positive and statistically significant Phase 3 SONICS and LOGICS studies, Strongbridge has submitted a New Drug Application to the U.S. Food and Drug Administration for RECORLEV® (levoketoconazole) for the treatment of endogenous Cushing’s syndrome,” said John H. Johnson, chief executive officer of Strongbridge Biopharma. “I am incredibly proud of our organization’s accomplishments over the last year and the team’s ability to execute against a number of strategic priorities that have set us up for long-term success. As we approach a number of transformational milestones in the year ahead, we remain focused on strengthening our organization through actively preparing for the potential approval and launch of RECORLEV, continued scientific exchange of results from the RECORLEV Phase 3 clinical program, and driving continued growth for KEVEYIS® (dichlorphenamide).” Corporate & Financial Highlights Rare Neuromuscular Franchise: KEVEYIS® (dichlorphenamide) Achieved KEVEYIS net product sales of $8.2 million for the fourth quarter ended, December 31, 2020, and $30.7 million for the full year ended December 31, 2020, exceeding its projected $28 million to $29 million guidance range, and representing a 41.5 percent increase over 2019 revenue of $21.7 million. Full-year 2021 revenue guidance of $34 million to $36 million for KEVEYIS. Rare Endocrine Franchise: RECORLEV® (levoketoconazole) On March 2, 2021, the Company announced it submitted a New Drug Application (NDA) for RECORLEV to the U.S. Food and Drug Administration; if approved, and assuming a projected 10-month review cycle, the launch of RECORLEV is anticipated in the first quarter of 2022. With regard to the clinical development program and medical affairs activities: In November, secondary endpoints results from the Phase 3 SONICS study of RECORLEV for the potential treatment of endogenous Cushing’s syndrome were published in the journal, Pituitary. Interim safety and efficacy results, including new data analyses, from the Phase 3 LOGICS study have been accepted for poster presentation at the Endocrine Society’s (ENDO) 2021 annual meeting, taking place virtually from March 20-23, 2021. Corporate and Financial Updates On February 16, 2021, the Company announced the promotion of Richard S. Kollender, who has served as chief operating officer of Strongbridge since September 2019, to president and chief financial officer. In this expanded role, Mr. Kollender will succeed Robert Lutz as chief financial officer and also maintain the responsibilities he held as chief operating officer. Mr. Kollender previously served on Strongbridge’s board of directors and also as chairman of the audit committee. These management changes are effective on March 3, 2021. Following completion in September 2020 of a $25 million equity raise and a recent year-end borrowing of an additional $10 million under the Company’s existing debt facility, Strongbridge reports approximately $87.5 million of cash and cash equivalents as of December 31, 2020. Assuming the availability and full draw-down of the remaining $10 million from its debt facility, the Company believes it can fund operations as currently planned into, and potentially beyond, the first quarter of 2023. Fourth Quarter 2020 Financial Results The Company’s net revenues from sales of KEVEYIS increased $2.6 million, or 47%, from $5.6 million for the three months ended December 31, 2019 to $8.2 million for the three months ended December 31, 2020. The Company recorded cost of sales of $0.4 million for the three months ended December 31, 2020, compared to cost of sales of $1.0 million for the same period in 2019. Cost of sales decreased due to changes in the assumptions underlying the allocation between the purchase price of our inventory and our supply agreement. Our gross margins were 95% for three months ended December 31, 2020, compared to gross margins of 82% for the same period in 2019. Selling, general and administrative expenses were $11.6 million for the three months ended December 31, 2020, compared to $12.0 million for the same period in 2019. The decrease during the current period was due to reduced personnel costs from headcount reductions and T&E savings from the prior period, partially offset by an increase in 3rd party expenses relating to our commercial activities. Research and development expenses were $5.3 million for the three months ended December 31, 2020, compared to $8.0 million for the same period in 2019. The decrease was primarily due to reduced support activities and materials for our LOGICS trial, offset by a small increase in costs from our OPTICS trial in 2020. For the three months ended December 31, 2020, basic net loss attributable to ordinary shareholders on a GAAP basis was ($11.9 million), or ($0.18) per share, compared to a basic net loss attributable to ordinary shareholders of ($9.0 million), or ($0.17) per share, for the same period in 2019. Net loss for the three months ended December 31, 2020 was higher than the same period in 2019 due to the prior period including income from the termination of a contract offset by an increase in net revenue in the current period. Additionally, there was a $2.3 million change in the revaluation of the fair value of our liability classified warrants recorded in 2020 compared to 2019 due to the increase in our stock price. For the three months ended December 31, 2020, non-GAAP basic net loss attributable to ordinary shareholders was ($7.6 million), or ($0.11) per share, compared to a non-GAAP basic net loss attributable to ordinary shareholders of ($13.5 million), or ($0.25) per share, for the same period in 2019. The decrease in non-GAAP net loss during the three months ended December 31, 2020 due to the increase in KEVEYIS revenues and overall lower expenses. Year-to-Date 2020 Financial Results The Company’s net revenues from sales of KEVEYIS increased $9.0 million, or 41.5%, from $21.7 million for the twelve months ended December 31, 2019 to $30.7 million for the twelve months ended December 31, 2020. The Company recorded cost of sales of $2.2 million for the twelve months ended December 31, 2020, compared to cost of sales of $3.8 million for the same period in 2019. Cost of sales decreased due to changes in the assumptions underlying the allocation between the purchase price of our inventory and our supply agreement. Our gross margins were 93% for twelve months ended December 31, 2020, compared to gross margins of 82% for the same period in 2019. Selling, general and administrative expenses were $40.9 million for the twelve months ended December 31, 2020, compared to $49.1 million for the same period in 2019. The decrease during the current period was due to reduced personnel costs from headcount reductions, reduced spending due to COVID-19, and lower third-party expenses. Additionally, the prior period had $3.2 million in one-time charges for severance expense. Research and development expenses were $25.8 million for the twelve months ended December 31, 2020, compared to $30.9 million for the same period in 2019. The spending reduction was primarily due to decreases in product development and supporting activities resulting from the completion of our SONICS trial in 2019 and higher costs related to our LOGICS trial in 2019, offset in part by an increase in costs from our OPTICS trial in 2020. For the twelve months ended December 31, 2020, basic net loss attributable to ordinary shareholders on a GAAP basis was ($45.1 million), or ($0.78) per share, compared to a basic net loss attributable to ordinary shareholders of ($49.5) million, or ($0.91) per share, for the same period in 2019. Net loss for the twelve months ended December 31, 2020 was lower than the same period in 2019 due to the increase in KEVEYIS revenue of $9.0 million and the reduction in selling, general and administrative and research and development expenses during the twelve months ended December 31, 2020 compared to the same period in 2019. Those decreases were offset by a $12.2 million change in the revaluation of the fair value of our liability classified warrants due to the increase in our stock price and income from termination of a contract which was recorded in the prior period. For the twelve months ended December 31, 2020, non-GAAP basic net loss attributable to ordinary shareholders was ($31.1 million), or ($0.54) per share, compared to a non-GAAP basic net loss attributable to ordinary shareholders of ($53.2 million), or ($0.98) per share, for the same period in 2019. The decrease in non-GAAP net loss during the twelve months ended December 31, 2020 was due to an increase in KEVEYIS revenue of $9.0 million and selling, general and administrative and research and development expenses decreasing during the twelve months ended December 31, 2020 compared to the same period in 2019. Conference Call Details Strongbridge will host a conference call on Wednesday, March 3, 2021 at 8:30 a.m. ET. To access the live call, dial (844) 285-7153 (domestic) or (478) 219-0180 (international) with conference ID 2452827. The conference call will also be webcast from the Company’s website at under the “Investor/Webcasts and Presentations” section. A replay of the call will be made available for one week following the conference call. To hear a replay of the call, dial (855) 859-2056 (domestic) or (404) 537-3406 (international) with conference ID 2452827. About Strongbridge Biopharma Strongbridge Biopharma is a global commercial-stage biopharmaceutical company focused on the development and commercialization of therapies for rare diseases with significant unmet needs. Strongbridge’s rare endocrine franchise includes RECORLEV® (levoketoconazole), a cortisol synthesis inhibitor currently being studied in Phase 3 clinical studies for the treatment of endogenous Cushing’s syndrome, and veldoreotide extended release, a preclinical next-generation somatostatin analog being investigated for the treatment of acromegaly and potential additional applications in other conditions amenable to somatostatin receptor activation. Both RECORLEV and veldoreotide have received orphan drug designation from the FDA and the European Medicines Agency. The Company’s rare neuromuscular franchise includes KEVEYIS® (dichlorphenamide), the first and only FDA-approved treatment for hyperkalemic, hypokalemic, and related variants of primary periodic paralysis. KEVEYIS has orphan drug exclusivity in the United States. About KEVEYIS KEVEYIS® (dichlorphenamide) is indicated for the treatment of primary hyperkalemic periodic paralysis, primary hypokalemic periodic paralysis, and related variants. In clinical studies, the most common side effects of KEVEYIS were a numbness or tingling, difficulty thinking and paying attention, changes in taste, and confusion. These are not all of the possible side effects that you may experience with KEVEYIS. Talk to your doctor if you have any symptoms that bother you or do not go away. You are encouraged to report side effects to Strongbridge Biopharma at 1-855-324-8912, or to the FDA at 1-800-FDA-1088 or visit . For additional KEVEYIS important safety information and the full prescribing information visit . About RECORLEV RECORLEV® (levoketoconazole) is an investigational cortisol synthesis inhibitor in development for the treatment of patients with endogenous Cushing’s syndrome, a rare but serious and potentially lethal endocrine disease caused by chronic elevated cortisol exposure. RECORLEV is the pure 2S,4R enantiomer of ketoconazole, a steroidogenesis inhibitor. RECORLEV has demonstrated in two successful Phase 3 studies to significantly suppress serum cortisol and has the potential to be a next-generation cortisol inhibitor. The Phase 3 program for RECORLEV includes SONICS and LOGICS: two multinational studies designed to evaluate the safety and efficacy of RECORLEV when used to treat endogenous Cushing’s syndrome. The SONICS study met its primary and secondary endpoints, demonstrating a statistically significant normalization rate of urinary free cortisol at six months. The LOGICS study, which met its primary endpoint, is a double-blind, placebo-controlled randomized-withdrawal study of RECORLEV that is designed to supplement the long-term efficacy and safety information supplied by SONICS. The ongoing long-term open label OPTICS study will gather further useful information related to the long-term use of RECORLEV. RECORLEV has received orphan drug designation from the FDA and the European Medicines Agency for the treatment of endogenous Cushing's syndrome. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the federal securities laws. The words “anticipate,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “project,” “target,” “will,” “would,” or the negative of these terms or other similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. All statements, other than statements of historical facts, contained in this press release, are forward-looking statements, including statements related to 2021 KEVEYIS revenue guidance, expected cash balances and cash runway, potential advantages of RECORLEV, the anticipated timing for the review of the NDA for RECORLEV and the potential launch of RECORLEV (if approved), Strongbridge’s strategy, plans, outcomes of product development efforts and objectives of management for future operations. Forward-looking statements involve risks and uncertainties that could cause actual results to differ materially from those expressed in such statement, including risks and uncertainties associated with clinical development and the regulatory approval process, the reproducibility of any reported results showing the benefits of RECORLEV, the adoption of RECORLEV by physicians, if approved, as treatment for any disease and the emergence of unexpected adverse events following regulatory approval and use of the product by patients. Additional risks and uncertainties relating to Strongbridge and its business can be found under the heading “Risk Factors” in Strongbridge’s Annual Report on Form 10-K for the year ended December 31, 2020 and its subsequent Quarterly Reports on Form 10-Q, as well as its other filings with the SEC. These forward-looking statements are based on current expectations, estimates, forecasts and projections and are not guarantees of future performance or development and involve known and unknown risks, uncertainties and other factors. The forward-looking statements contained in this press release are made as of the date of this press release, and Strongbridge Biopharma does not assume any obligation to update any forward-looking statements except as required by applicable law. Contacts: Corporate and Media Relations Elixir Health Public Relations Lindsay Rocco +1 862-596-1304 lrocco@elixirhealthpr.com Investor Relations Solebury Trout Mike Biega +1 617-221-9660 mbiega@soleburytrout.com