Dinutuximab beta

Consolidated net revenue of € 1,743.1 million in the first nine months, +12.0% or +9.3% on a like-for-like basis(3) and at constant exchange rates (CER)EBITDA(1) of € 665.7 million, +11.8%, revenue margin of 38.2% Adjusted net income(2) of € 445.4 million, +9.5% Net income of € 338.4 million, +11.1% Free cash flow(4) at € 434.3 million, +€ 42.5 million vs prior yearNet debt(5) at € 1,317.3 million, just below 1.6x EBITDAFinancial targets for FY 2024 confirmed, excluding any potential contribution from Enjaymo® Global Rare Diseases business to be further strengthened by announced agreement with Sanofi to acquire the global rights to Enjaymo®; deal expected to close by end of 2024, subject to regulatory approvals Isturisa®approved in China for the treatment of adult patients with Cushing syndromeResolution to distribute an interim 2024 dividend of € 0.60 per shareApproval of a new share buy-back program to service the stock option and performance shares plans Milan, November 8th, 2024 – The Board of Directors of Recordati S.p.A. approved the Group’s Interim Report on 30th September 2024, representing additional voluntary financial reporting (6). The Report was prepared using the assessment, measurement and recognition criteria prescribed by international accounting standards (IFRS). The Group’s Interim Report dated 30th September 2024 will be available on 11th November at the company’s offices and on the company’s website (www.recordati.com) and can also be viewed on the authorized storage system 1Info (www.1Info.it). Rob Koremans, Chief Executive Officer of Recordati, commented: “During the first nine months, we delivered double-digit revenue and profit growth, reflecting the strong performance across the business with more patients benefiting from our therapies. These results reflect the continued commitment of all our people, who are instrumental to our success. With a positive outlook for the remainder of the year, we are well positioned to achieve our upgraded financial objectives for FY 2024. Together, we will continue to build on our momentum and create lasting value for our stakeholders. “I am also pleased to highlight our recent agreement with Sanofi to acquire the global rights to Enjaymo®, the only approved treatment option for patients with cold agglutinin disease. This transaction will reinforce our strong commitment to serving patients with rare diseases and is a strong strategic fit to our portfolio, bringing a product with a robust profile, supported by a strong team, with attractive financial contributions expected on both the top and bottom lines.” Financial highlights Consolidated net revenue for the first nine months of 2024 was € 1,743.1 million, up 12.0% versus the first nine months of 2023 or 9.3% on a like-for-like(3) basis at CER. This was driven by strong business momentum across both Specialty & Primary Care and Rare Diseases. The adverse FX impact for the first nine months of 2024 was € 35.9 million (-2.3%), mainly driven by the devaluation of the Turkish lira, particularly in the first and third quarters, which was compensated by high price inflation in Türkiye. Specialty & Primary Care revenue totaled € 1,094.4 million in the first nine months of 2024, growing 11.1% or 6.5% on a like-for-like(3) basis at CER (+2.6% excluding Türkiye). This reflects a particularly strong performance from the Urology franchise with 51.4% growth versus the previous year, driven by the double-digit growth of Eligard® and the € 82.9 million contribution of Avodart® and Combodart®/Duodart®(7) (versus € 3.8 million in the third quarter of 2023). The Cardiovascular and Gastrointestinal franchises remained resilient and delivered slight growth, while the Cough and Cold business reflected solid in-market performance in the context of a softer flu season as compared to the previous year.Rare Diseases revenue totaled € 605.6 million in the first nine months of 2024, up 14.1% as compared to the first nine months of 2023, or 14.5% at CER, driven by the key growth franchises of Oncology and Endocrinology which continue to show substantial further growth potential. The Endocrinology franchise achieved net revenue of € 239.4 million, an increase of 36.0% and reflecting the continued strong performance of Isturisa® and double-digit growth of Signifor®. The Oncology franchise achieved net revenue of € 175.9 million, an increase of 17.1%, driven by double-digit growth from both Qarziba® and Sylvant®. The Metabolic franchise achieved net revenue of € 190.2 million, a decrease of 6.9%, mainly due to generic competition for Carbaglu® in the US and EMEA, with growth in other international markets. Adjusted operating income(8) was € 539.5 million for the first nine months of 2024, up 9.7% over the previous year, and 31.0% of net revenue versus 31.6% in the same period last year. Operating income was € 504.1 million in the first nine months of 2024, up 14.9% over the first nine months of 2023, absorbing gross margin-related non-cash charges of € 28.1 million (versus € 47.2 million in first nine months of 2023), arising from the unwind of the fair value step up of the acquired rare oncology inventory. Non-recurring costs were € 7.3 million in the first nine months of 2024, versus € 5.4 million in the first nine months of 2023, which includes € 2.5 million related to the agreement with Sanofi for the global rights to Enjaymo®.EBITDA(1) was € 665.7 million for the first nine months of 2024, up 11.8% compared to the first nine months of 2023, with margin of 38.2% of net revenue, in line with the previous year. Strong revenue and operating leverage were, in part, offset by a reduction in adjusted gross profit margin due to the consolidation of Avodart® and Combodart®/ Duodart® and product mix.Financial expenses were € 62.3 million, up by € 13.3 million compared to the previous year, including € 2.8 million in FX losses (mostly unrealized, compared to losses of € 0.3 million in the first nine months of 2023) and € 3.9 million of net monetary losses from hyperinflation accounting (compared to a gain of € 1.8 million in the first nine months of 2023). Adjusted net income(2) was € 445.4 million, 25.6% of revenue, up by 9.5% compared to the same period of 2023, with higher adjusted operating income partially offset by an increase in financial expenses and a higher tax rate, following a statutory tax rate increase in some countries. Net income was € 338.4 million, 19.4% of revenue, an increase of 11.1% versus first nine months of 2023, with the higher tax rate and financing expenses offsetting the higher operating income. Free cash flow(4) was € 434.3 million for the first nine months of 2024, an increase of € 42.5 million versus the the first nine months of 2023, driven by higher EBITDA which was slightly offset by higher interests and income taxes paid. Net debt(5) as of 30th September 2024 was € 1,317.3 million, or leverage of just below 1.6x EBITDA(6), compared to net debt of € 1,579.4 million on 31st December 2023. Shareholders’ equity was € 1,876.1 million. Pipeline and Corporate Development The Isturisa® new drug application (NDA) was approved by the China National Medical Products Administration (NMPA) in September for the treatment of adult patients with Cushing syndrome. On October 4th, the Group announced an agreement with Sanofi to acquire the global rights to Enjaymo®, a biologic which is the only approved targeted product for the treatment of cold agglutinin disease (CAD), a rare B-cell lymphoproliferative disorder. In 2022, Enjaymo® was granted approval by the U.S. Food and Drug Administration (FDA), the European Commission (EC) and the Japanese Ministry of Health, Labor and Welfare. Enjaymo® generated approximately € 100 million in revenue over the last 12 months as of August 2024 and is expected to generate revenue in excess of € 150 million in FY 2025, with peak sales potential of € 250-300 million. The transaction is expected to be immediately accretive at the EBITDA level, with margin above the current Rare Diseases average as of 2025. Under the terms of the agreement, Recordati will make an upfront payment of US$ 825 million and additional commercial milestone payments of up to US$ 250 million if net sales reach certain thresholds at or above the top end of peak year sales expectations. The transaction is expected to close by the end of 2024, subject to regulatory clearances (see also press release issued on October 4th 2024). The deal will be funded by existing cash and new committed bank debt facilities. Business outlook Thanks to continued strong momentum, the Group is on track to deliver the targets for FY 2024 as adjusted upward on July 30th (excluding any potential contribution from Enjaymo®): Net revenue between € 2,300 and € 2,340 millionEBITDA(1) between € 845 and € 865 million; margin of +/- 37%Adjusted net income(2) between € 560 and € 580 million; margin of +/- 24.5%Minimal contribution expected from Enjaymo®, subject to timing of regulatory approvals Additional resolutions Interim dividend 2024 The Board of Directors passed a resolution to distribute an interim 2024 dividend to shareholders of € 0.60 (gross of tax withholdings) per share outstanding at the ex-dividend date, thus excluding treasury stock held in the Company’s portfolio on that date. The 2024 interim dividend will be payable as of 20th November 2024 (record date 19th November 2024), with coupon no. 34, to shareholders registered on 18th November 2024. The Independent Auditor EY S.p.A. has issued the opinion required by Art. 2433 – bis, paragraph 5 of the Italian Civil Code, which is available at the company’s offices. The Directors’ Report and Recordati S.p.A. financial statements as of 30th June 2024, based on which the latter’s Board of Directors resolved the distribution of the interim dividend, are available at the company’s office and website (www.recordati.com), and can also be viewed on the authorized storage system 1Info (www.1Info.it). Limited assurance on sustainability reporting In compliance with the Italian Legislative Decree No. 125/2024 – transposing in Italy the EU Directive No. 2022/2464 on Corporate Sustainability Reporting (so called “CSRD”) – the Board of Directors, upon proposal of the Board of Statutory Auditors, has resolved to integrate – starting from this financial year 2024 - the mandate for activities regarding the limited assurance attestation on sustainability reporting, set forth by the aforementioned decree, to Ernst & Young S.p.A., by extending to such activities the current engagement granted by the Shareholders Meeting held on 29th April 2020 with respect to the attestation of the non-financial disclosure. Appointment of the new Financial Reporting Officer pursuant to art.154-bis of TUF The Board of Directors has appointed, until revocation and upon favourable opinion of the Board of Statutory Auditors Niccolo Giovannini - VP Group Finance, reporting to Luigi La Corte, Group Chief Financial Officer - as Financial Reporting Officer pursuant to art.154-bis of TUF and art.25 of the Articles of Association. The appointment is effective as of today, following the approval of Group’s Interim Report on 30th September 2024. Approval of a new share buy-back program to service the stock option and performance shares plans Today, the Board of Directors has also approved the launch of a new share buy-back program under art. 5 of (EU) Regulation no. 596/2014 for a maximum of 1,500,000 ordinary shares with a maximum cash outlay of € 90,000,000. This implements the resolution adopted by the Shareholders’ meeting held on 22nd April 2024 to purchase Recordati ordinary shares in order to service current and future stock option/performance shares plans in favour of the Recordati Group’s management or share-based incentive plans that might be approved by the Company in the future. All details required by the applicable regulations on the launch of the share buy-back program - whose start is envisaged on 12th November 2024 until 15th April 2025, following the end of the current buyback programme envisaged by 11th November 2024 as announced on 9th May 2024 - will be included in a specific press release that will be issued according to law and to which reference is made. (1) Net income before income taxes, financial income and expenses, depreciation, amortization and write-downs of property, plant and equipment, intangible assets and goodwill, non-recurring items and non-cash charges arising from the allocation of the purchase price of EUSA Pharma to the gross margin of acquired inventory according to IFRS 3. (2) Net income excluding the amortization and write-down of intangible assets (except software) and goodwill, non-recurring items, non-cash charges arising from the allocation of the purchase price of EUSA Pharma to the gross margin of acquired inventory pursuant to IFRS 3, and monetary net gains/losses from hyperinflation (IAS 29), net of tax effects. (3) Pro-forma growth calculated excluding revenue of Avodart® and Combodart®/ Duodart® for both 2024 and 2023.(4) Total cash flow excluding financing items, milestones, dividends, purchases of treasury shares net of proceeds from exercise of stock options.(5) Cash and cash equivalents, less bank debts and loans, which include the measurement at fair value of hedging derivatives. (6) Please note that Italian Legislative Decree 25/2016, which implements Directive 2013/50/EU, no longer stipulates the submission of an interim management report, which was previously required in terms of paragraph 5 of Art. 154- ter of Italian Legislative Decree 58/1998.(7) Trademarks are owned by or licensed to the GSK group of companies. Transition of commercialization effectively concluded.(8) Net income before income taxes, financial income and expenses and non-recurring items, non-cash charges arising from the allocation of the purchase price of EUSA Pharma to the gross margin of acquired inventory according to IFRS 3. Conference Call Recordati will host a conference call today, November 8th at 4:00 p.m. CET (3:00 p.m. GMT) to present the results for the first nine months of 2024. The dial-in numbers for the conference call service are: Italy + 39 02 802 09 11, toll free 800 231 525UK + 44 1 212818004, toll free (44) 0 800 0156371USA +1 718 7058796, toll free (1) 1 855 2656958France +33 1 70918704Germany +49 6917415712 Participants are invited to dial in 10 minutes before conference time. If conference operator assistance is required to connect, please dial *0. The slides that will be referenced during the call will be available at www.recordati.com under Investors/Company Presentations. The audio conference live webcast will also be available at the following link Recordati is an international pharmaceutical group listed on the Italian Stock Exchange (XMIL: REC), with roots dating back to a family-run pharmacy in Northern Italy in the 1920s. We are uniquely structured to provide treatments across specialty and primary care, and rare diseases. Our fully integrated operations span clinical development, chemical and finished product manufacturing, commercialization and licensing. We operate in approximately 150 countries across EMEA, the Americas and APAC with over 4,450 employees. We believe that health is a fundamental right, not a privilege. Today, our purpose of “unlocking the full potential of life” aims at empowering individuals to live life to the fullest, whether addressing common health challenges or the rarest. Investor Relations Eugenia Litz +44 7824 394 750 investorelations@recordati.itGianluca Saletta +39 348 979 4876 investorelations@recordati.itMedia Relations ICR Healthcare US:Alexis Feinberg +1 203 939 2225 recordatiuspr@westwicke.com UK, Europe & Rest of World:Jessica Hodgson +44 7561 424 788recordati@consilium-comms.com This document contains forward-looking statements relating to future events and future operating, economic and financial results of the Recordati group. By their nature, forward-looking statements involve risk and uncertainty because they depend on the occurrence of future events and circumstances. Actual results may therefore differ materially from those forecast for a variety of reasons, most of which are beyond the Recordati group’s control. The information on the pharmaceutical specialties and other products of the Recordati group contained in this document is intended solely as information on the activities of the Recordati Group, and, as such, it is not intended as a medical scientific indication or recommendation, or as advertising. RECORDATI GROUP Summary of the consolidated results,prepared in accordance with International Financial Reporting Standards (IFRS)(€ thousands) INCOME STATEMENTFirst nine months 2024First nine months 2023Change %NET REVENUE1,743,0811,556,17412.0Cost of sales(556,171)(490,495)13.4GROSS PROFIT1,186,9101,065,67911.4Selling expenses(360,709)(345,506)4.4Research and development expenses(204,849)(182,239)12.4General and administrative expenses(110,014)(93,630)17.5Other income/(expenses), net(7,240)(5,553)30.4OPERATING INCOME504,098438,75114.9Financial income/(expenses), net(62,319)(49,054)27.0PRE-TAX INCOME441,779389,69713.4Income taxes(103,379)(85,205)21.3NET INCOME 338,400304,49211.1Adjusted gross profit (1)1,214,9861,113,1679.1Adjusted operating income (2)539,518491,6089.7Adjusted net income (3)445,361406,5669.5EBITDA (4)665,666595,57311.8Net income attributable to: Equity holders of the Parent338,400304,49211.1Non-controlling interests00n.s.EARNINGS PER SHARE (euro) Basic(5)1.6401.48110.7Diluted(6)1.6181.45611.1(1) Gross profit adjusted from impact of non-cash charges arising from the allocation of the purchase price of EUSA Pharma to the gross margin of acquired inventory according to IFRS 3. (2) Net income before income taxes, financial income and expenses, non-recurring items and non-cash charges arising from the allocation of the purchase price of EUSA Pharma to the gross margin of acquired inventory according to IFRS 3.(3) Net income excluding amortization and write-downs of intangible assets (except software) and goodwill, non-recurring items, non-cash charges arising from the allocation of the purchase price of EUSA Pharma to the gross margin of acquired inventory (IFRS 3) and monetary net gains/losses from hyperinflation (IAS 29), net of tax effects.(4) Net income before income taxes, financial income and expenses, depreciation, amortization and write-downs of property, plant and equipment, intangible assets and goodwill, non-recurring items and non-cash charges arising from the allocation of the purchase price of EUSA Pharma to the gross margin of acquired inventory according to IFRS 3.(5) Earnings per share (EPS) are based on average shares outstanding during the respective period, 206.299.160 in 2024 and 205.421.410 in 2023. These amounts are calculated deducting treasury shares in the portfolio, the average of which was 2.825.996 shares in 2024 and 3.703.746 shares in 2023.(6) Diluted earnings per share is calculated by taking into account rights granted to employees.COMPOSITION OF NET REVENUEFirst nine months 2024First nine months 2023Change % Total revenue1,743,0811,556,17412.0 Italy258,631234,30410.4 International1,484,4501,321,87012.3 RECORDATI GROUP(€ thousands) Reconciliation of Net income to EBITDA(1) First nine months 2024First nine months 2023Net income338,400304,492Income taxes103,37985,205Financial income/(expenses), net62,31949,054Non-recurring expenses7,3445,369Non-cash charges from PPA inventory uplift28,07647,488Adjusted operating income(2)539,518491,608Depreciation, amortization and write-downs126,148103,965EBITDA(1)665,666595,573 Reconciliation of Net income to Adjusted Net income(3) First nine months 2024First nine months 2023Net income338,400304,492Amortization and write-downs of intangible assets(excluding software)100,15781,180Tax effect(22,619)(17,405)Non-recurring operating expenses7,3445,369Tax effect(1,943)(1,340)Non-cash charges from PPA inventory uplift28,07647,488Tax effect(7,019)(11,881)Monetary net (gain)/losses from hyperinflation (IAS29)3,900(1,759)Tax effect(935)422Adjusted net income(3)445,361406,566 (1) Net income before income taxes, financial income and expenses, depreciation, amortization and write-downs of property, plant and equipment, intangible assets and goodwill, non-recurring items and non-cash charges arising from the allocation of the purchase price of EUSA Pharma to the gross margin of acquired inventory (IFRS 3).(2) Net income before income taxes, financial income and expenses, non-recurring items and non-cash charges arising from the allocation of the purchase price of EUSA Pharma to the gross margin of acquired inventory (IFRS 3).(3) Net income excluding amortization and write-downs of intangible assets (except software) and goodwill, non-recurring items, non-cash charges arising from the allocation of the purchase price of EUSA Pharma to the gross margin of acquired inventory (IFRS 3) and monetary net gains/losses from hyperinflation (IAS 29), net of tax effects. RECORDATI GROUPSummary of the consolidated results, prepared in accordance with International Financial Reporting Standards (IFRS)(€ thousands) ASSETS30.09.202431.12.2023Property, plant and equipment187,848178,657Intangible assets1,847,7041,938,197Goodwill789,123778,350Other equity investments and securities16,86521,555Other non-current assets13,87112,458Deferred tax assets92,56776,674TOTAL NON-CURRRENT ASSETS2,947,9783,005,891 Inventories410,107404,831Trade receivables474,514445,193Other receivables102,46699,401Other current assets26,78219,924Derivative instruments measured at fair value6,71011,079Cash and cash equivalents235,020221,812TOTAL CURRENT ASSETS1,255,5991,202,240TOTAL ASSETS4,203,5774,208,131 RECORDATI GROUPSummary of the consolidated results, prepared in accordance with International Financial Reporting Standards (IFRS)(€ thousands) EQUITY AND LIABILITIES30.09.202431.12.2023 Share capital26,14126,141Share premium reserve83,71983,719Treasury shares(139,881)(127,970)Reserve for derivative instruments(2,180)(286)Translation reserve(301,627)(264,700)Other reserves61,53761,219Profits carried forward1,809,9971,636,451Net income338,400389,214Interim dividend0(117,396)Shareholders’ equity attributable to equity holders of the Parent1,876,1061,686,392Shareholders’ equity attributable to non-controlling interests00TOTAL SHAREHOLDERS’ EQUITY1,876,1061,686,392 Loans - due after one year1,264,8841,353,216Provisions for employee benefits21,40521,239Deferred tax liabilities134,488144,208TOTAL NON-CURRENT LIABILITIES1,420,7771,518,663 Trade payables276,907263,979Other payables198,213174,407Tax liabilities100,73867,110Other current liabilities4,6165,307Provisions for risks and charges19,24116,596Derivative instruments measured at fair value14,21219,993Loans - due within one year270,337355,752Short-term debts to banks and other lenders22,43099,932TOTAL CURRENT LIABILITIES906,6941,003,076TOTAL SHAREHOLDERS’ EQUITY AND LIABILITIES4,203,5774,208,131 RECORDATI GROUPSummary of consolidated results prepared in accordance with International Financial Reporting Standards (IFRS) (€ thousands) CASH FLOW STATEMENTFirst nine months 2024First nine months 2023OPERATING ACTIVITIES Net income338,400304,492Income taxes103,37985,206Net interest54,41848,158Depreciation of property, plant and equipment24,00321,577Amortization of intangible assets97,59182,304Write-downs4,55484Equity-settled share-based payment transactions10,1207,515Other non-monetary components41,06955,772Change in other assets and other liabilities(11,985)(20,538)Cash flow generated/(used) by operating activities before change in working capital661,549584,570Change in: - inventories(41,813)(31,681)- trade receivables(36,418)(73,753)- trade payables14,22330,082Change in working capital(64,008)(75,352)Interest received4,0073,923Interest paid(64,284)(50,314)Income taxes paid(82,634)(53,282)Cash flow generated/(used) by operating activities454,630409,545INVESTMENT ACTIVITIES Investments in property, plant and equipment(21,743)(17,998)Disposals of property, plant and equipment1,385329Investments in intangible assets(15,377)(345,597)Disposals of intangible assets2,351287Sale of non-current assets held for sale2,0003,000Cash flow generated/(used) by investment activities(31,384)(359,979)FINANCING ACTIVITIES Opening of loans144,872348,256Repayment of loans(320,185)(214,701)Payment of lease liabilities(8,311)(8,116)Change in short-term debts to banks and other lenders(71,722)(45,008)Dividends paid(130,220)(129,071)Purchase of treasury shares(78,087)(6,483)Sale of treasury shares52,74419,681Cash flow generated/(used) by financing activities(410,909)(35,442)Change in cash and cash equivalents12,33714,124Opening cash and cash equivalents221,812284,734Currency translation effect8713,422Closing cash and cash equivalents235,020302,280 DECLARATION BY THE FINANCIAL REPORTING OFFICERThe Financial Reporting Officer, Luigi La Corte, declares, pursuant to paragraph 2 of Article 154-bis of the Consolidated Law on Finance, that the accounting information contained in this press release corresponds to the Company’s documentation, books and accounting records.

对于企业，其实不必过于担心审评通过率多少，更应该关注自己的产品是否满足医生的临床治疗需求，是否有效解决了患者的病痛，是否有造福民众的高度社会责任感。关于这一系列命题，太阳升医药的经验或许能给行业带来些许启发。 又是一年国谈季。 9月19日，国家医保局召开了国家医保药品目录调整拟谈判/竞价企业沟通会后，行业里许多人的心都悬到了嗓子眼——据传，今年国家医保药品目录调整中，通过形式审查的目录外品种，专家评审环节通过率与前两年相比有了明显下降。 持续的医药寒冬里，国谈入口又收紧，难免让不少业内人陷入了悲观。 悲观可以理解，但必须认识到，近两年，国谈权重更多向创新倾斜，保障越来越多好药走进了千家万户。医保局和专家评审的根本目的，其实也是在医保控费的基础上鼓励有价值的创新。 明确了这一点，再反推逻辑，就不难认识到许多药品进不了国谈的根本原因：要么创新性不足，没有满足临床需求；要么成本太高，缺乏经济性。 因此对企业来说，与其抱怨，不如更多关心自身产品价值，思考如何顺应医保局的精神，针对性制定战略战术，既能实现创新与可及性的平衡，又能借助政策实现产品的放量。 01 产品布局，必须顺应政策精神 有关专家评审环节通过率，目前业内传闻纷纷，有的说60%以下，有的说还不到50%。 可无论具体比率究竟是多少，行业都应该始终把握住一个基本点：医保局的根本目的，就是在医保控费的基础上，鼓励真正意义上的创新。 从这一点出发，专家对临床数据、试验数据等的真实可靠性要求越来越严，也是顺理成章。 此外对于老生常谈的“进院难”问题，相关部门也一直在推进解决，部门间的政策存在着联动。 比如今年7月底，上海市医保局联合7部门印发了《上海市进一步完善多元支付机制支持创新药械发展的若干措施》，专门提到了国谈药的可及性问题。 渠道方面，对进院难、处方难等的问题，文件规定开通创新药挂网“绿色通道”，督促医疗机构做到“应配尽配”；调整优化医疗机构考核机制，医院不得以用药目录数量、“药/耗占比”等原因限制创新药械入院”；对谈判纳入国家医保目录的创新药前三年实行单列预算，不纳入当年医院医保总额预算等。 控费方面，文件还规定“在DRG/DIP支付方面对创新药械予以倾斜，提高新技术应用病例支付标准，新技术应用高倍率病例不设控制比例，成规模新技术应用可单独成组”。 从多方面出发，一套组合拳打下去，既严格管控又大力扶持，将创新优质的国谈最快推向市场。 而近五年，我国国谈药品支出一直在持续增长，2023年较2022年多支付了17.1%，说明有限的医保资金正在快速向创新药物倾斜。 总体来看，不难得出结论：国家对支持新药好药、鼓励真创新的态度从来没有变过。但企业在研发和构建产品组合时，也必须发掘临床价值，找到市场需求和经济性之间的平衡。 可以说每一次国谈，背后都是对企业产品自身价值的考验。只有真正具有价值的产品，才能得到政策的助力。 以赛诺菲的度普利尤单抗为例，其2020年经国家谈判纳入医保目录，受益于“双通道”管理政策，覆盖面迅速由2021Q1的7个省增加到了2021Q2的14个省，仅一个季度就翻了倍。 度普利尤单抗为什么能乘东风起飞？根本原因还是具有真正的临床价值。这是FDA批准的首个治疗特应性皮炎的生物制品，其上市前，AD患者只能以糖皮质激素、免疫抑制剂来进行缓解，要被迫承受巨大的副作用。因此副作用较低的度普利尤单抗一经上市，就受到了患者的大力追捧。 太阳升医药开发了国内首个针对更年期综合症的创新药黄母颗粒。我国约1.6亿更年期女性，超过1.2亿女性深受更年期综合症的困扰, 严重影响生活和工作质量。临床医生和患者对激素替代疗法的副作用都存在诸多顾虑，患者难以维持有效治疗。关黄母颗粒的开发和上市弥补了更年期综合征女性的治疗需求，发扬中医药的治疗优势，聚焦优势病种，关黄母颗粒有效缓解更年期综合症患者出现的烘热出汗、失眠焦虑、易怒、骨痛、乏力等症状。 曾有专家评价，目前早期已上市的大多数中成药临床适应症宽泛，缺乏明确的疾病名称，给临床医生使用带来困扰，也增加药事服务的难度，导致临床价值难以评估。但关黄母颗粒的临床定位与适应症明确聚焦疾病，指导临床用药。 创新属性强，有临床价值，经济性高，使关黄母颗粒迅速得到了各级医保部门的认可。该药2018年通过中药新药评审，是当年国家药监局体制改革后首个获批的中药新药，并进入了2021年的医保谈判目录。 2023年8月，广东省医保局发布《关于做好医保药品单独支付保障工作的通知》，将包含关黄母颗粒等403个品种纳入单独支付的品种范围，自当年9月1日起实施，试行期限2年。 按文件相关要求，名单中药品费用由统筹基金与定点医疗机构单列结算，不纳入相关额度；参保人在定点医疗机构普通门诊就医发生的单独支付药品费用，不设起付线，不纳入门诊统筹按人头包干额度，不单独设立最高支付限额；参保人在住院、急诊留院观察、日间病房等发生的单独支付药品费用，也由统筹基金单独支付，不列入该次住院的医疗总费用核算范围。 随着太阳升医药持续开展专业化学术推广，加强临床医生对患者和疾病的关注，提倡合理用药，加强患者教育提升对疾病认知积极开展治疗，并努力提升患者用药的可及性，关黄母颗粒不仅在国内得到了普遍认可，还走出了国门，陆续在非洲加纳、南苏丹、赞比亚等国家获批上市。 02 激烈竞争下，企业应尽早构建合理的产品组合 顺应政策精神的基础上，行业也必须要接受一个事实：当下的竞争，正变得越来越激烈。 首当其冲的是，今年通过形式审查的目录外品种数量达到了249个。反观2023年和2022年，这个数字分别为224个、198个，参与谈判和竞价的药品数量分别为143个和147个，专家评审通过率在60%和70%左右。 基数变大，竞争形势自然就恶化了，专家评审通过率下降不足为奇。 而这249个品种里，恒瑞、中生等大公司都在跑马竞逐，比如恒瑞医药有6个品种药物在列，包括尼莫地平口服溶液、富马酸泰吉利定注射液等；中国生物制药有5款药物，如富马酸安奈克替尼胶囊、派安普利单抗注射液等. Biopharma中，百济神州-U的达妥昔单抗β注射液、注射用贝林妥欧单抗，康方生物的PD-1/CTLA-4卡度尼利双抗和PD-1/VEGF依沃西双抗亦均通过了专家评审环节。 最后，复星凯特、科济药业、药明巨诺等多家CAR-T产品等多个热门品种也都出现在了初审名单中。不过由于定价居高不下，依然很可能上不了谈判桌，又“陪跑”了一程。 整体看下来，目录外药物虽然大多都具有很强的创新属性，但也存在不少靶点和适应症重复的现象，进一步加剧了内卷。 正因此，药企在布局管线和产品时，不光要强调创新，也要同时充分考虑到市场需求、临床价值和同业竞争问题。 E药经理人注意到，本次医保谈判目录内的196个品种，其中不少具有优秀的临床价值，等待在本次国谈中得到进一步发掘。比如这一次的申报条件2限定为：今年底协议到期，适应症或功能主治未发生重大变化，因适应症与医保支付范围不一致，主动申请调整支付范围的谈判药品。 这一条款的意义在于，许多已经进入了国谈的药品，随着真实世界应用越来越多，更多适应症场景得以开辟，政策支持也与时俱进，随着场景范围的扩大而进一步增加。 门冬氨酸钾镁木糖醇注射液就是一个很好的案例。临床上，补钾制剂多为注射剂，介于快速补钾的效果，要求其溶媒为葡萄糖，但对于有补钾需求的糖尿病患者带来风险。针对基数庞大的糖尿病患者群体，如何安全有效的补钾是临床治疗中需要关注的问题。 了解市场需求和痛点后，门冬氨酸钾镁木糖醇注射液独创性地采取了木糖醇作为溶媒，并申请了独家专利。临床上，这款药能惠及广大低血钾症、肾病、手术后恢复期的糖尿病患者，让临床医生在治疗中终于不必再“左右为难”。 该药独特的临床价值，不仅很快被临床医生们意识到，广泛应用起来，亦迅速得到了医保部门的关注。2023年12月13日，国家医保局正式发布《国家基本医疗保险、工伤保险和生育保险药品目录（2023年）》，关黄母颗粒、门冬氨酸钾镁木糖醇注射液成功纳入，并于今年1月1日起正式执行。 技术上，换一个溶媒并不困难。难得之处在于，首先要有一双发现患者需求的眼睛。 这款药物同样出自太阳升医药的手笔。然而，除了关黄母颗粒、门冬氨酸钾镁木糖醇注射液外，在太阳升医药产品目录中还可以看到二十余款具有独家专利、原研进口、医保集采等属性资质的药品，这不得不引起行业猜想：太阳升医药究竟是一家生么样的企业？随着对太阳升医药创始人方小东先生从业经历的深挖细品，答案也就慢慢水清石见了。 太阳升集团北京研发中心 方小东，曾任主板上市公司珍宝岛药业事业部经理、总经理、总裁，在执掌珍宝岛药业长达15年的时间里，珍宝岛市场营销从最初的千万级不断跨越到10亿、20亿、50亿规模，营销模式被行业权威研究机构收录为“十大经典案例”。方小东个人荣获“中国十大医药精英人物”称号。 综上不难看出，太阳升医药专业且独到的选品眼光是基因使然。值得一提的是，太阳升除了在选品上坚持以临床需求为导向，致力于发掘广大患者未被满足的需要，还实现了中药创新药、生物医药的协同发展。 为了进一步发力创新，公司2019年在安徽亳州市落地后，组建了由强大的院士专家顾问和30名博士、博士后组成的核心科研团队，已获授权发明专利31项，为接下来的长远发展夯实了基础。 太阳升医药的成功经验，给当下行业里的许多玩家都带来了启示。 当下阶段，靶点和适应症内卷依然不可避免，这一点是由我国企业全球新FIC相对不足，follow产品居多的现状决定的，属于结构性问题。但相关部门的态度，又始终是鼓励“真创新”，从未改变。 面对这一看似矛盾的局面，对于中小企业来说，最好的战略不是像大公司一样，去在高度内卷的适应症和靶点上抢时间、砸资本，走Pharma路线，而是坚持从临床需求出发，进一步发掘那些未被满足的适应症，走差异化路线打造产品矩阵。等到先发产品被广大患者接受，有了商业回报后，再进一步扩大规模，向Pharma看齐。 这其实也是过去几年的医药寒冬里企业界、资本界、科学界逐渐达成的共识：与其盲目跟风，重复建设，不如专注手上已有资源的整合和优化，打破惯性思维，回归商业最本质的常识，回到市场，回到价值创造。 一切并不复杂。 一审| 黄佳 二审| 李芳晨 三审| 李静芝 精彩推荐 CM10 | 集采 | 国谈 | 医保动态 | 药审 | 人才 | 薪资 | 榜单 | CAR-T | PD-1 | mRNA | 单抗 | 商业化 | 国际化 ｜ 猎药人系列专题 | 出海 启思会 | 声音·责任 | 创百汇 | E药经理人理事会 | 微解药直播 | 大国新药 | 营销硬观点 | 投资人去哪儿 | 分析师看赛道 | 药事每周谈 | 医药界·E药经理人 | 中国医药手册 创新100强榜单 | 恒瑞 | 中国生物制药 | 百济 | 石药 | 信达 | 君实 | 复宏汉霖 ｜翰森 | 康方生物 | 上海医药 | 和黄医药 | 东阳光药 |  荣昌 | 亚盛医药 | 齐鲁制药 | 康宁杰瑞 | 贝达药业 | 微芯生物 | 复星医药 ｜再鼎医药｜亚虹医药 跨国药企50强榜单 | 辉瑞 | 艾伯维 | 诺华 | 强生 | 罗氏 | BMS | 默克 | 赛诺菲 | AZ | GSK | 武田 | 吉利德科学 | 礼来 | 安进 | 诺和诺德 | 拜耳 | 莫德纳 | BI | 晖致 | 再生元