Triptorelin acetate

Ipsen delivers robust sales growth in the first quarter of 2023 and confirms its full-year guidance Paris (France), 27 April 2023Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-driven biopharmaceutical company, today presents its sales performance for the first quarter of 2023. Q1 2023 Q1 2022 % change €m €m Actual CER1 Oncology 570.8 556.4 2.6% 1.1% Neuroscience 156.4 120.2 30.2% 24.4% Rare Disease 14.7 11.3 29.8% 29.0% Total Sales2 741.9 687.9 7.8% 5.7% Highlights Total-sales growth of 5.7% at CER1, or 7.8% as reported, driven by the performance of the growth platforms3, up by 14.7%1, with Dysport® (abobotulinumtoxinA) up by 25.2%1 and Cabometyx® (cabozantinib) up by 31.0%1, respectively. The performance included contributions from newly acquired Tazverik® (tazemetostat) and Bylvay® (odevixibat)Completion of the definitive merger agreement for the acquisition of Albireo, expanding Ipsen’s scope in Rare DiseaseRegulatory-decision dates in the U.S. confirmed for Bylvay in Alagille syndrome and palovarotene in fibrodysplasia ossificans progressiva (FOP), respectivelyFull-year 2023 guidance confirmed, with total-sales growth greater than 4.0% at CER1 and a core operating margin of around 30% of total sales David Loew, Chief Executive Officer, commented:“Ipsen continues to make excellent progress in its transformation. We delivered further robust sales growth in the quarter, led by the standout performances of Dysport and Cabometyx. Based on the continued sales momentum, we are confirming our guidance for the full year. I was also delighted by the further enhancement of our pipeline, portfolio and organization as a result of the recent acquisition of Albireo; through our global presence, we will continue to meet the unmet medical needs of an increasing number of patients. As we replenish the pipeline and execute on recent transactions, we look forward to several milestones for our business, including the Phase III data readout for elafibranor and anticipated regulatory developments for Onivyde, palovarotene and Bylvay.” Full-year 2023 guidanceIpsen has confirmed its financial guidance for FY 20234: Total-sales growth greater than 4.0%, at constant exchange rates. Based on the average level of exchange rates in Q1 2023, an adverse impact on total sales of around 2% from currencies is expectedCore operating margin around 30% of total sales, excluding any potential impact of incremental investments from future external-innovation transactions Business development In March 2023, Ipsen announced that it had completed the acquisition of Albireo Pharma, Inc., a leading innovator in bile-acid modulators to treat rare liver conditions. Ipsen acquired all issued and outstanding shares at a price of $42.00 per share in cash, plus one non-transferable contingent value right of $10.00 per share. Pipeline development In February 2023, it was announced that the U.S. Food and Drug Administration had accepted the supplemental New Drug Application for a second Bylvay indication, for patients with Alagille syndrome. The administration also issued a Prescription Drug User Fee Act (PDUFA) action date of 15 June 2023. In March 2023, it was announced that the PDUFA action date in the U.S., for the resubmitted New Drug Application for palovarotene as a potential treatment for FOP, will be 16 August 2023. The Company also recently requested a re-examination of the negative opinion from the CHMP5 for palovarotene, received in January 2023. Conference callA conference call and webcast for investors and analysts will begin today at 2pm, Paris time. Participants can access the call and its details by registering here; webcast details can be found here. A recording will be available on ipsen.com. CalendarIpsen intends to publish its half-year and second-quarter results on 27 July 2023. Notes All financial figures are in € millions (€m). The performance shown in this announcement covers the three-month period to 31 March 2023 (the first quarter or Q1 2023), compared to the three-month period to 31 March 2022 (Q1 2022), unless stated otherwise. Ipsen Ipsen is a global, mid-sized biopharmaceutical company focused on transformative medicines in Oncology, Rare Disease and Neuroscience. With total sales of €3.0bn in FY 2022, Ipsen sells medicines in over 100 countries. Alongside its external-innovation strategy, the Company’s research and development efforts are focused on its innovative and differentiated technological platforms located in the heart of leading biotechnological and life-science hubs: Paris-Saclay, France; Oxford, U.K.; Cambridge, U.S.; Shanghai, China. Ipsen has around 5,400 colleagues worldwide and is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information, visit ipsen.com. Contacts Investors Craig MarksVice President, Investor Relations+44 (0)7584 349 193 Media Amy WolfVice President and Head of Corporate Brand Strategy and Communications+41 79 576 07 23 Ioana PiscociuSenior Manager, Global Media Relations+33 6 69 09 12 96 Total sales by therapy area and medicine A breakdown of medicine sales by geographical area is shown in the appendix. Q1 2023 Q1 2022 % change €m €m Actual CER6 Oncology 570.8 556.4 2.6% 1.1% Somatuline® 263.2 286.0 -8.0% -9.8% Cabometyx 130.4 98.9 31.9% 31.0% Decapeptyl 130.0 129.2 0.6% 0.8% Onivyde 36.9 40.1 -7.9% -12.3% Tazverik 9.2 - n/a n/a Other Oncology 1.0 2.2 -54.0% -53.6% Neuroscience 156.4 120.2 30.2% 24.4% Dysport 154.6 118.4 30.5% 25.2% Other Neuroscience 1.8 1.7 4.5% -18.0% Rare Disease 14.7 11.3 29.8% 29.0% Bylvay 5.0 - n/a n/a NutropinAq® 5.4 7.1 -24.1% -24.0% Increlex® 4.2 4.2 -0.6% -3.2% Total Sales 741.9 687.9 7.8% 5.7% Somatuline® (lanreotide): in North America, limited sales erosion of 2.7%7, with adverse U.S. pricing partly offset by a favourable impact reflecting the reduced level of U.S wholesaler-buying levels in Q1 2022. In Europe, sales declined by 25.7%7, driven by the impact of generic lanreotide in a number of markets, including France, Spain and Italy. Sales in the Rest of the World increased by 7.3%7, a result of solid performances in several geographies, including Latin AmericaDecapeptyl: performance impacted by reduced sales in China, reflecting the prior-year benefit from increased stocking in anticipation of COVID-19 lockdown restrictions, partly offset by continuous market-share uptakes across a number of geographiesCabometyx: performance reflected strong volume uptakes in renal cell carcinoma across most geographies, as a second-line monotherapy and as a first-line therapy in combination with nivolumab in more countriesOnivyde: performance impacted by phasing of shipments to Ipsen’s ex-U.S. partner, partly offset by the solid underlying growth in the U.S.Tazverik: sales consolidated for the full quarter, following the completion of the acquisition of Epizyme in August 2022. Commercial sales growing by 21% year on year7,8Dysport: performance driven by further growth in the aesthetics market, reflected in increased sales to Ipsen’s partner, Galderma, and strong demand in most therapeutics markets Bylvay: sales consolidated for one month, following the acquisition of Albireo that was completed in March 2023 Total sales by geographical area Q1 2023 Q1 2022 % change €m €m Actual CER7 North America 244.8 226.4 8.1% 3.6% Europe9 296.3 304.7 -2.7% -2.3% Rest of the World 200.8 156.9 28.0% 23.2% Total Sales 741.9 687.9 7.8% 5.7% North America: sales grew by 3.6%7, driven by a solid performance from Onivyde and the contribution from newly acquired medicines Tazverik and Bylvay, partly offset by reduced sales of Dysport to Galderma in aesthetics, as well as the gradual erosion of SomatulineEurope: sales declined by 2.3%7, mainly driven by the performance of Somatuline that reflected the impact of generic lanreotide and by reduced Onivyde sales to Ipsen’s ex-U.S. partner, offset by strong growth of Cabometyx, as well as Dysport in the therapeutics and aesthetics marketsRest of the World: sales grew by 23.2%7, driven by a solid Cabometyx performance and Dysport, primarily in Latin America, the Middle East and North Africa Appendix: geographic breakdown of total sales by medicine Total North America Europe Rest of the World Q1 2023 Q1 2022 % change Q1 2023 Q1 2022 % change Q1 2023 Q1 2022 % change Q1 2023 Q1 2022 % change €m €m Actual CER10 €m €m Actual CER10 €m €m Actual CER10 €m €m Actual CER10 Oncology 570.8 556.4 2.6% 1.1% 202.9 184.3 10.1% 5.7% 239.8 267.9 -10.5% -10.0% 128.0 104.3 22.8% 19.9% Somatuline 263.2 286.0 -8.0% -9.8% 153.7 151.5 1.4% -2.7% 77.4 105.5 -26.7% -25.7% 32.1 29.0 10.9% 7.3% Cabometyx 130.4 98.9 31.9% 31.0% 4.2 4.0 4.8% 6.9% 87.1 75.2 15.7% 16.3% 39.2 19.7 99.1% 85.8% Decapeptyl 130.0 129.2 0.6% 0.8% - - - - 73.3 73.7 -0.5% 0.1% 56.7 55.5 2.2% 1.6% Onivyde 36.9 40.1 -7.9% -12.3% 35.7 28.7 24.7% 19.1% 1.2 11.4 -89.5% -90.9% - - - - Tazverik 9.2 - n/a n/a 9.2 - n/a n/a - - - - - - - - Other Oncology 1.0 2.2 -54.0% -53.6% 0.1 0.1 50.8% 53.8% 0.9 2.0 -55.9% -55.8% 0.0 0.1 n/a n/a Neuroscience 156.4 120.2 30.2% 24.4% 36.5 39.5 -7.5% -11.9% 47.7 28.4 68.1% 69.6% 72.2 52.3 38.0% 29.0% Dysport 154.6 118.4 30.5% 25.2% 36.5 39.5 -7.5% -11.9% 47.7 28.4 68.1% 69.6% 70.3 50.5 39.1% 31.0% Other Neuroscience 1.8 1.7 4.5% -18.0% - - - - - - - - 1.8 1.7 4.5% -18.0% Rare Disease 14.7 11.3 29.8% 29.0% 5.3 2.6 n/a 95.6% 8.7 8.4 4.0% 4.2% 0.6 0.3 n/a n/a Bylvay 5.0 - n/a n/a 3.0 - n/a n/a 2.0 - n/a n/a 0.0 - n/a n/a NutropinAq 5.4 7.1 -24.1% -24.0% - - - - 5.1 6.8 -25.0% -24.9% 0.3 0.3 -2.3% -1.5% Increlex 4.2 4.2 -0.6% -3.2% 2.3 2.6 -10.2% -14.1% 1.5 1.5 -2.8% -2.1% 0.3 0.0 n/a n/a Total Sales 741.9 687.9 7.8% 5.7% 244.8 226.4 8.1% 3.6% 296.3 304.7 -2.7% -2.3% 200.8 156.9 28.0% 23.2% Forward-looking statements The forward-looking statements, objectives and targets contained herein are based on Ipsen’s management strategy, current views and assumptions. Such statements involve known and unknown risks and uncertainties that may cause actual results, performance or events to differ materially from those anticipated herein. All of the above risks could affect Ipsen’s future ability to achieve its financial targets, which were set assuming reasonable macroeconomic conditions based on the information available today. Use of the words ‘believes’, ‘anticipates’ and ‘expects’ and similar expressions are intended to identify forward-looking statements, including Ipsen’s expectations regarding future events, including regulatory filings and determinations. Moreover, the targets described in this document were prepared without taking into account external-growth assumptions and potential future acquisitions, which may alter these parameters. These objectives are based on data and assumptions regarded as reasonable by Ipsen. These targets depend on conditions or facts likely to happen in the future, and not exclusively on historical data. Actual results may depart significantly from these targets given the occurrence of certain risks and uncertainties, notably the fact that a promising medicine in early development phase or clinical trial may end up never being launched on the market or reaching its commercial targets, notably for regulatory or competition reasons. Ipsen must face or might face competition from generic medicine that might translate into a loss of market share. Furthermore, the research and development process involves several stages each of which involves the substantial risk that Ipsen may fail to achieve its objectives and be forced to abandon its efforts with regards to a medicine in which it has invested significant sums. Therefore, Ipsen cannot be certain that favorable results obtained during preclinical trials will be confirmed subsequently during clinical trials, or that the results of clinical trials will be sufficient to demonstrate the safe and effective nature of the medicine concerned. There can be no guarantees a medicine will receive the necessary regulatory approvals or that the medicine will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Other risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and healthcare legislation; global trends toward healthcare cost containment; technological advances, new medicine and patents attained by competitors; challenges inherent in new-medicine development, including obtaining regulatory approval; Ipsen's ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of Ipsen’s patents and other protections for innovative medicines; and the exposure to litigation, including patent litigation, and/or regulatory actions. Ipsen also depends on third parties to develop and market some of its medicines which could potentially generate substantial royalties; these partners could behave in such ways which could cause damage to Ipsen’s activities and financial results. Ipsen cannot be certain that its partners will fulfil their obligations. It might be unable to obtain any benefit from those agreements. A default by any of Ipsen’s partners could generate lower revenues than expected. Such situations could have a negative impact on Ipsen’s business, financial position or performance. Ipsen expressly disclaims any obligation or undertaking to update or revise any forward-looking statements, targets or estimates contained in this press release to reflect any change in events, conditions, assumptions or circumstances on which any such statements are based, unless so required by applicable law. Ipsen’s business is subject to the risk factors outlined in its registration documents filed with the French Autorité des Marchés Financiers. The risks and uncertainties set out are not exhaustive and the reader is advised to refer to Ipsen’s 2021 Universal Registration Document, available on ipsen.com. 1 At constant exchange rates (CER), which exclude any foreign-exchange impact by recalculating the performance for the relevant period by applying the exchange rates used for the prior period. 2 Total sales in this announcement are unaudited IFRS consolidated sales.3 Dysport, Decapeptyl® (triptorelin), Cabometyx and Onivyde® (irinotecan).4 The performance of Consumer HealthCare, divested in July 2022, has been excluded from all commentary and comparisons to prior performance.5 The Committee for Medicinal Products for Human Use, the European Medicines Agency's committee responsible for human medicines.6 At CER, which excludes any foreign-exchange impact by recalculating the performance for the relevant period by applying the exchange rates used for the prior period.7 At CER, which excludes any foreign-exchange impact by recalculating the performance for the relevant period by applying the exchange rates used for the prior period.8 Reference to Epizyme’s published Q1 2022 performance.9 Defined in this announcement as the E.U., the U.K., Iceland, Liechtenstein, Norway and Switzerland.10 At CER, which excludes any foreign-exchange impact by recalculating the performance for the relevant period by applying the exchange rates used for the prior period. Attachment Ipsen - Q1 2023 sales-results announcement

Credit: Shutterstock/ Chaos810 Earlier this week, the Florida Senate advanced a bill to restrict gender-affirming care for transgender minors. The bill is one of many that are currently being discussed in different states. Free Report How is the Biopharmaceutical industry evolving? 2021 was a year of continued innovation and change in the Biopharmaceutical industry. As the COVID-19 pandemic continues to take its toll on businesses worldwide, it’s time to look for new ways to create value, prepare for the future, and remain competitive in the ever-changing landscape. GlobalData’s expansive report examines the business environment and trends that shape the Biopharmaceutical industry. We highlight the most impactful emerging technologies, as well as the industry, regulatory, and macroeconomic factors that influence growth prospects. Access the report to: Benchmark the impact of major themes on the Biopharmaceutical industry. Gain a deeper "on the ground" perspective through exclusive opinions and analysis from industry respondents. Evaluate the effects of COVID-19 on the sector. Download the full report to understand what to expect and how to align your strategies for success. By GlobalData Submit Country Code UK (+44) USA (+1) Algeria (+213) Andorra (+376) Angola (+244) Anguilla (+1264) Antigua & Barbuda (+1268) Argentina (+54) Armenia (+374) Aruba (+297) Australia (+61) Austria (+43) Azerbaijan (+994) Bahamas (+1242) Bahrain (+973) Bangladesh (+880) Barbados (+1246) Belarus (+375) Belgium (+32) Belize (+501) Benin (+229) Bermuda (+1441) Bhutan (+975) Bolivia (+591) Bosnia Herzegovina (+387) Botswana (+267) Brazil (+55) Brunei (+673) Bulgaria (+359) Burkina Faso (+226) Burundi (+257) Cambodia (+855) Cameroon (+237) Canada (+1) Cape Verde Islands (+238) Cayman Islands (+1345) Central African Republic (+236) Chile (+56) China (+86) Colombia (+57) Comoros (+269) Congo (+242) Cook Islands (+682) Costa Rica (+506) Croatia (+385) Cuba (+53) Cyprus North (+90392) Cyprus South (+357) Czech Republic (+42) Denmark (+45) Djibouti (+253) Dominica (+1809) Dominican Republic (+1809) Ecuador (+593) Egypt (+20) El Salvador (+503) Equatorial Guinea (+240) Eritrea (+291) Estonia (+372) Ethiopia (+251) Falkland Islands (+500) Faroe Islands (+298) Fiji (+679) Finland (+358) France (+33) French Guiana (+594) French Polynesia (+689) Gabon (+241) Gambia (+220) Georgia (+7880) Germany (+49) Ghana (+233) Gibraltar (+350) Greece (+30) Greenland (+299) Grenada (+1473) Guadeloupe (+590) Guam (+671) Guatemala (+502) Guinea (+224) Guinea - Bissau (+245) Guyana (+592) Haiti (+509) Honduras (+504) Hong Kong (+852) Hungary (+36) Iceland (+354) India (+91) Indonesia (+62) Iran (+98) Iraq (+964) Ireland (+353) Israel (+972) Italy (+39) Jamaica (+1876) Japan (+81) Jordan (+962) Kazakhstan (+7) Kenya (+254) Kiribati (+686) Korea North (+850) Korea South (+82) Kuwait (+965) Kyrgyzstan (+996) Laos (+856) Latvia (+371) Lebanon (+961) Lesotho (+266) Liberia (+231) Libya (+218) Liechtenstein (+417) Lithuania (+370) Luxembourg (+352) Macao (+853) Macedonia (+389) Madagascar (+261) Malawi (+265) Malaysia (+60) Maldives (+960) Mali (+223) Malta (+356) Marshall Islands (+692) Martinique (+596) Mauritania (+222) Mayotte (+269) Mexico (+52) Micronesia (+691) Moldova (+373) Monaco (+377) Mongolia (+976) Montserrat (+1664) Morocco (+212) Mozambique (+258) Myanmar (+95) Namibia (+264) Nauru (+674) Nepal (+977) Netherlands (+31) New Caledonia (+687) New Zealand (+64) Nicaragua (+505) Niger (+227) Nigeria (+234) Niue (+683) Norfolk Islands (+672) Northern Marianas (+670) Norway (+47) Oman (+968) Palau (+680) Panama (+507) Papua New Guinea (+675) Paraguay (+595) Peru (+51) Philippines (+63) Poland (+48) Portugal (+351) Puerto Rico (+1787) Qatar (+974) Reunion (+262) Romania (+40) Russia (+7) Rwanda (+250) San Marino (+378) Sao Tome & Principe (+239) Saudi Arabia (+966) Senegal (+221) Serbia (+381) Seychelles (+248) Sierra Leone (+232) Singapore (+65) Slovak Republic (+421) Slovenia (+386) Solomon Islands (+677) Somalia (+252) South Africa (+27) Spain (+34) Sri Lanka (+94) St. Helena (+290) St. Kitts (+1869) St. Lucia (+1758) Sudan (+249) Suriname (+597) Swaziland (+268) Sweden (+46) Switzerland (+41) Syria (+963) Taiwan (+886) Tajikstan (+7) Thailand (+66) Togo (+228) Tonga (+676) Trinidad & Tobago (+1868) Tunisia (+216) Turkey (+90) Turkmenistan (+7) Turkmenistan (+993) Turks & Caicos Islands (+1649) Tuvalu (+688) Uganda (+256) Ukraine (+380) United Arab Emirates (+971) Uruguay (+598) Uzbekistan (+7) Vanuatu (+678) Vatican City (+379) Venezuela (+58) Vietnam (+84) Virgin Islands - British (+1284) Virgin Islands - US (+1340) Wallis & Futuna (+681) Yemen (North)(+969) Yemen (South)(+967) Zambia (+260) Zimbabwe (+263) Country UK USA Afghanistan Åland Islands Albania Algeria American Samoa Andorra Angola Anguilla Antarctica Antigua and Barbuda Argentina Armenia Aruba Australia Austria Azerbaijan Bahamas Bahrain Bangladesh Barbados Belarus Belgium Belize Benin Bermuda Bhutan Bolivia Bonaire, Sint Eustatius and Saba Bosnia and Herzegovina Botswana Bouvet Island Brazil British Indian Ocean Territory Brunei Darussalam Bulgaria Burkina Faso Burundi Cambodia Cameroon Canada Cape Verde Cayman Islands Central African Republic Chad Chile China Christmas Island Cocos Islands Colombia Comoros Congo Democratic Republic of the Congo Cook Islands Costa Rica Côte d"Ivoire Croatia Cuba Curaçao Cyprus Czech Republic Denmark Djibouti Dominica Dominican Republic Ecuador Egypt El Salvador Equatorial Guinea Eritrea Estonia Ethiopia Falkland Islands Faroe Islands Fiji Finland France French Guiana French Polynesia French Southern Territories Gabon Gambia Georgia Germany Ghana Gibraltar Greece Greenland Grenada Guadeloupe Guam Guatemala Guernsey Guinea Guinea-Bissau Guyana Haiti Heard Island and McDonald Islands Holy See Honduras Hong Kong Hungary Iceland India Indonesia Iran Iraq Ireland Isle of Man Israel Italy Jamaica Japan Jersey Jordan Kazakhstan Kenya Kiribati North Korea South Korea Kuwait Kyrgyzstan Lao Latvia Lebanon Lesotho Liberia Libyan Arab Jamahiriya Liechtenstein Lithuania Luxembourg Macao Macedonia, The Former Yugoslav Republic of Madagascar Malawi Malaysia Maldives Mali Malta Marshall Islands Martinique Mauritania Mauritius Mayotte Mexico Micronesia Moldova Monaco Mongolia Montenegro Montserrat Morocco Mozambique Myanmar Namibia Nauru Nepal Netherlands New Caledonia New Zealand Nicaragua Niger Nigeria Niue Norfolk Island Northern Mariana Islands Norway Oman Pakistan Palau Palestinian Territory Panama Papua New Guinea Paraguay Peru Philippines Pitcairn Poland Portugal Puerto Rico Qatar Réunion Romania Russian Federation Rwanda Saint Helena, Ascension and Tristan da Cunha Saint Kitts and Nevis Saint Lucia Saint Pierre and Miquelon Saint Vincent and The Grenadines Samoa San Marino Sao Tome and Principe Saudi Arabia Senegal Serbia Seychelles Sierra Leone Singapore Slovakia Slovenia Solomon Islands Somalia South Africa South Georgia and The South Sandwich Islands Spain Sri Lanka Sudan Suriname Svalbard and Jan Mayen Swaziland Sweden Switzerland Syrian Arab Republic Taiwan Tajikistan Tanzania Thailand Timor-Leste Togo Tokelau Tonga Trinidad and Tobago Tunisia Turkey Turkmenistan Turks and Caicos Islands Tuvalu Uganda Ukraine United Arab Emirates US Minor Outlying Islands Uruguay Uzbekistan Vanuatu Venezuela Vietnam British Virgin Islands US Virgin Islands Wallis and Futuna Western Sahara Yemen Zambia Zimbabwe Kosovo Submit By clicking the Download Free Report button, you accept the terms and conditions and acknowledge that your data will be used as described in the GlobalData privacy policy By downloading this Report, you acknowledge that we may share your information with our white paper partners/sponsors who may contact you directly with information on their products and services. Visit our privacy policy for more information about our services, how we may use, process and share your personal data, including information on your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address. Thank you. Please check your email to download the Report. Many politicians have cited doubts about the potential “harm” caused by puberty blockers and hormone therapies associated with gender-affirming care and concerns about incorrect treatment as major factors. However, health specialists refute most safety concerns for the use of drug classes as part of gender affirming care, citing years of research and care. Dr. Barry DeCoster, PhD, an associate professor of bioethics at the University of Michigan, describes gender affirming care as “safe and effective” for most patients. Most drugs used here are prescribed off-label, and experts say pharmaceutical companies remain hesitant to conduct clinical trials studying gender-affirming care. This has pushed clinicians to explore different routes to prove the validity of these drugs. The World Health Organization (WHO) defines gender-affirming care as a range of social, psychological, behavioural, and medical interventions that are “designed to support and affirm an individual’s gender identity” when it conflicts with the gender they were assigned at birth. The laws proposed in several bills range in their potential influence; from restricting the prescription of gender-affirming drugs to criminalising the use of such drugs to allowing states to pursue lawsuits against parents who allow children to access such treatments. Dr Lisa Campo-Engelstein, a bioethicist, predicts that these factors and subsequent restrictions on statewide healthcare coverage will heavily restrict patient access to gender-affirming care. Campo-Engelstein is the director of the Institute for Bioethics & Health Humanities at the University of Texas Medical Branch, Galveston. With restricted access and reduced prescription of puberty blockers and hormone therapies, the impact on pharmaceutical companies manufacturing these therapies also remains unclear. Standards of care for gender-affirming therapies The global standards for gender-affirming medical practices stem from the World Professional Association for Transgender Health (WPATH)’s yearly guidelines . WPATH’s recommendations are based on “the best available science and expert professional consensus” to give “safe and effective pathways” for transsexual, transgender, and gender-nonconforming people . WPATH suggests an individualised approach to treating gender dysphoria. Generally, individuals first take on social gender-affirmation changes. Dr Sean Cahill, PhD, the associate editor of the global, peer-reviewed journal LGBT Health, says “for most young people, healthcare provided doesn’t even go beyond that [social affirmation of gender identity], but a lot of people… are misrepresenting what gender affirming care looks like in this country”. Following this, they may progress to the use of puberty blockers. After a minor has been on puberty blockers for two to three years, they may have the option to use hormone therapies upon further evaluation. Puberty blockers are used in gender affirming care to delay the onset of puberty for gender dysphoric children of any gender. These drugs do not tend to have permanent effects and only cause a pause in puberty. While hormone therapies are allowed in the US, the age limit for this is dependent on the geographical area. Standard puberty blockers include Gonadotrophin Releasing Hormone (GnRH) analogues like Trelstar (triptorelin), Supprelin (Histrelin), and Zoladex (Goserelin), are only prescribed off label in the US and UK for gender affirming care. For example, Ontario, Canada-based Verity Pharmaceuticals’s Trelstar was FDA approved for palliative treatment of advanced prostate cancer and the other two are indicated for treating central precocious puberty and endometriosis, respectively. Several companies that produce commonly used puberty blockers such as Vantas Pharmaceuticals, Endo International and TerSera Therapeutics, have remained silent on the ongoing debate. Additionally, hormone therapies—testosterone to “masculinise” and/or transition to men, and oestrogen for transitioning to women and/or “feminising” the body—are an integral part of the plan. Testosterone stops menstrual cycles, decreases the ovaries’ ability to make oestrogen and more, while oestrogen allows individuals to develop breasts, rounder hips and changes to the reproductive system. The most commonly used testosterones in the US include Endo International’s Delatestryl (testosterone enanthate) and Aveed (testosterone undecanoate), and Pfizer’s Depo-testosterone (testosterone cypionate). These changes can be irreversible and can take up to five years to reach maximum effect. Common oestrogen therapies are mostly oestradiols such as Novartis’ Vivelle-Dot, Ascend Therapeutics’ Estrogel, and Teva Pharmaceuticals’ Gynodiol. Oestrogen hormone therapies are often approved for restoring hormone levels for cisgendered women, to reduce symptoms such as hot flushes and vaginal dryness. Dr. Edmund Horowicz PhD, a lecturer in medical law and ethics at the University of Liverpool, counters discourse from those such as the Norwegian Healthcare Investigation Board UKOM , that characterises puberty blockers as “experimental” and untrustworthy due to their off-label prescription. One of the reasons why organisations, including pharma companies, do not enter this debate is because these drugs are used off label, says Horowicz. As these drugs are not licensed for the specific indication of gender-affirming care, collecting safety data on off-label use is not viewed as a priority. Cahill says health centers across the nation, like his, see a sizable transgender population, and would be able to easily conduct longitudinal drug studies investigating safety measures and patient satisfaction. But the funding is not there, he adds. The onus for funding would most likely be on government agencies like the National Institutes of Health (NIH), says Cahill. Moreover, there are some logistical barriers in designing such studies. While it is important to run randomised placebo-controlled trials, giving participants sham gender affirming care, for example, would worsen mental distress associated with gender dysphoria, says Horowicz. There might also be ethical considerations for denying care and some subjects may also drop out of studies. Still, while the long-term impact of puberty blockers on bone-density and muscular skeletal development still needs to be studied, Horowicz asserts this should be viewed as a risk associated with a drug, similar to all other drugs. In Georgia’s bill , the legislators reason that the bill would be necessary under the “do no harm” principle, expressing safety concerns for minors. Due to a lack of long-term data into the possible impacts of these drugs, some people have proposed a possible solution of only prescribing these drugs within studies until more data arises. But this would restrict these drugs to only those who choose to be trial participants, creating further ethical conundrums, says Horowicz. Furthermore, Horowicz states that clinical frameworks, such as WPATH’s guidelines, are already based on evidence from trials of these well-established drugs. New studies for this use would be “more to satisfy those who are concerned about the provision of puberty blockers than any kind of traditional expected model of pharmacological research”. Possible consequences Last year, the Biden administration proposed the implementation of a rule in section 1557 , that would protect against limiting access to care by stating that “categorically refusing to provide treatment to an individual based on their gender identity is prohibited discrimination”. However, the recent state-specific gender affirmation legislations may look to contradict this. For example, in Florida, the Agency for Health Care Administration has prohibited Medicaid from covering any kind of gender-affirming care procedures like hormone therapies. Campo-Engelstein says some states like Texas don’t allow coverage for gender-affirming care through public systems. However, she points out that gender affirmation practices are still completely legal in the context of intersex “correction”, yet lawmakers contradict their use for transgender individuals. An immediate consequence of the possible legal ramifications of the bills is a shift in patient care and healthcare coverage, particularly for publicly funded hospitals. A bellwether for which states may see major changes in drug prescription and coverage, may be the ones that saw changes in access to abortion following the Supreme Court overturning Roe v Wade . “States that are banning abortion are going to be more likely to ban gender affirming care, because they’re both motivated by the same social and political values,” says Campo-Engelstein. DeCoster says the lack of consultation with clinicians and patients in deciding these legislations does a disservice to those most affected by the new laws. “We should be having a health care system that is helping support and to raise up people to give them what they need,” he says. Free Report How is the Biopharmaceutical industry evolving? 2021 was a year of continued innovation and change in the Biopharmaceutical industry. As the COVID-19 pandemic continues to take its toll on businesses worldwide, it’s time to look for new ways to create value, prepare for the future, and remain competitive in the ever-changing landscape. GlobalData’s expansive report examines the business environment and trends that shape the Biopharmaceutical industry. We highlight the most impactful emerging technologies, as well as the industry, regulatory, and macroeconomic factors that influence growth prospects. Access the report to: Benchmark the impact of major themes on the Biopharmaceutical industry. Gain a deeper "on the ground" perspective through exclusive opinions and analysis from industry respondents. Evaluate the effects of COVID-19 on the sector. Download the full report to understand what to expect and how to align your strategies for success. By GlobalData Submit Country Code UK (+44) USA (+1) Algeria (+213) Andorra (+376) Angola (+244) Anguilla (+1264) Antigua & Barbuda (+1268) Argentina (+54) Armenia (+374) Aruba (+297) Australia (+61) Austria (+43) Azerbaijan (+994) Bahamas (+1242) Bahrain (+973) Bangladesh (+880) Barbados (+1246) Belarus (+375) Belgium (+32) Belize (+501) Benin (+229) Bermuda (+1441) Bhutan (+975) Bolivia (+591) Bosnia Herzegovina (+387) Botswana (+267) Brazil (+55) Brunei (+673) Bulgaria (+359) Burkina Faso (+226) Burundi (+257) Cambodia (+855) Cameroon (+237) Canada (+1) Cape Verde Islands (+238) Cayman Islands (+1345) Central African Republic (+236) Chile (+56) China (+86) Colombia (+57) Comoros (+269) Congo (+242) Cook Islands (+682) Costa Rica (+506) Croatia (+385) Cuba (+53) Cyprus North (+90392) Cyprus South (+357) Czech Republic (+42) Denmark (+45) Djibouti (+253) Dominica (+1809) Dominican Republic (+1809) Ecuador (+593) Egypt (+20) El Salvador (+503) Equatorial Guinea (+240) Eritrea (+291) Estonia (+372) Ethiopia (+251) Falkland Islands (+500) Faroe Islands (+298) Fiji (+679) Finland (+358) France (+33) French Guiana (+594) French Polynesia (+689) Gabon (+241) Gambia (+220) Georgia (+7880) Germany (+49) Ghana (+233) Gibraltar (+350) Greece (+30) Greenland (+299) Grenada (+1473) Guadeloupe (+590) Guam (+671) Guatemala (+502) Guinea (+224) Guinea - Bissau (+245) Guyana (+592) Haiti (+509) Honduras (+504) Hong Kong (+852) Hungary (+36) Iceland (+354) India (+91) Indonesia (+62) Iran (+98) Iraq (+964) Ireland (+353) Israel (+972) Italy (+39) Jamaica (+1876) Japan (+81) Jordan (+962) Kazakhstan (+7) Kenya (+254) Kiribati (+686) Korea North (+850) Korea South (+82) Kuwait (+965) Kyrgyzstan (+996) Laos (+856) Latvia (+371) Lebanon (+961) Lesotho (+266) Liberia (+231) Libya (+218) Liechtenstein (+417) Lithuania (+370) Luxembourg (+352) Macao (+853) Macedonia (+389) Madagascar (+261) Malawi (+265) Malaysia (+60) Maldives (+960) Mali (+223) Malta (+356) Marshall Islands (+692) Martinique (+596) Mauritania (+222) Mayotte (+269) Mexico (+52) Micronesia (+691) Moldova (+373) Monaco (+377) Mongolia (+976) Montserrat (+1664) Morocco (+212) Mozambique (+258) Myanmar (+95) Namibia (+264) Nauru (+674) Nepal (+977) Netherlands (+31) New Caledonia (+687) New Zealand (+64) Nicaragua (+505) Niger (+227) Nigeria (+234) Niue (+683) Norfolk Islands (+672) Northern Marianas (+670) Norway (+47) Oman (+968) Palau (+680) Panama (+507) Papua New Guinea (+675) Paraguay (+595) Peru (+51) Philippines (+63) Poland (+48) Portugal (+351) Puerto Rico (+1787) Qatar (+974) Reunion (+262) Romania (+40) Russia (+7) Rwanda (+250) San Marino (+378) Sao Tome & Principe (+239) Saudi Arabia (+966) Senegal (+221) Serbia (+381) Seychelles (+248) Sierra Leone (+232) Singapore (+65) Slovak Republic (+421) Slovenia (+386) Solomon Islands (+677) Somalia (+252) South Africa (+27) Spain (+34) Sri Lanka (+94) St. Helena (+290) St. Kitts (+1869) St. Lucia (+1758) Sudan (+249) Suriname (+597) Swaziland (+268) Sweden (+46) Switzerland (+41) Syria (+963) Taiwan (+886) Tajikstan (+7) Thailand (+66) Togo (+228) Tonga (+676) Trinidad & Tobago (+1868) Tunisia (+216) Turkey (+90) Turkmenistan (+7) Turkmenistan (+993) Turks & Caicos Islands (+1649) Tuvalu (+688) Uganda (+256) Ukraine (+380) United Arab Emirates (+971) Uruguay (+598) Uzbekistan (+7) Vanuatu (+678) Vatican City (+379) Venezuela (+58) Vietnam (+84) Virgin Islands - British (+1284) Virgin Islands - US (+1340) Wallis & Futuna (+681) Yemen (North)(+969) Yemen (South)(+967) Zambia (+260) Zimbabwe (+263) Country UK USA Afghanistan Åland Islands Albania Algeria American Samoa Andorra Angola Anguilla Antarctica Antigua and Barbuda Argentina Armenia Aruba Australia Austria Azerbaijan Bahamas Bahrain Bangladesh Barbados Belarus Belgium Belize Benin Bermuda Bhutan Bolivia Bonaire, Sint Eustatius and Saba Bosnia and Herzegovina Botswana Bouvet Island Brazil British Indian Ocean Territory Brunei Darussalam Bulgaria Burkina Faso Burundi Cambodia Cameroon Canada Cape Verde Cayman Islands Central African Republic Chad Chile China Christmas Island Cocos Islands Colombia Comoros Congo Democratic Republic of the Congo Cook Islands Costa Rica Côte d"Ivoire Croatia Cuba Curaçao Cyprus Czech Republic Denmark Djibouti Dominica Dominican Republic Ecuador Egypt El Salvador Equatorial Guinea Eritrea Estonia Ethiopia Falkland Islands Faroe Islands Fiji Finland France French Guiana French Polynesia French Southern Territories Gabon Gambia Georgia Germany Ghana Gibraltar Greece Greenland Grenada Guadeloupe Guam Guatemala Guernsey Guinea Guinea-Bissau Guyana Haiti Heard Island and McDonald Islands Holy See Honduras Hong Kong Hungary Iceland India Indonesia Iran Iraq Ireland Isle of Man Israel Italy Jamaica Japan Jersey Jordan Kazakhstan Kenya Kiribati North Korea South Korea Kuwait Kyrgyzstan Lao Latvia Lebanon Lesotho Liberia Libyan Arab Jamahiriya Liechtenstein Lithuania Luxembourg Macao Macedonia, The Former Yugoslav Republic of Madagascar Malawi Malaysia Maldives Mali Malta Marshall Islands Martinique Mauritania Mauritius Mayotte Mexico Micronesia Moldova Monaco Mongolia Montenegro Montserrat Morocco Mozambique Myanmar Namibia Nauru Nepal Netherlands New Caledonia New Zealand Nicaragua Niger Nigeria Niue Norfolk Island Northern Mariana Islands Norway Oman Pakistan Palau Palestinian Territory Panama Papua New Guinea Paraguay Peru Philippines Pitcairn Poland Portugal Puerto Rico Qatar Réunion Romania Russian Federation Rwanda Saint Helena, Ascension and Tristan da Cunha Saint Kitts and Nevis Saint Lucia Saint Pierre and Miquelon Saint Vincent and The Grenadines Samoa San Marino Sao Tome and Principe Saudi Arabia Senegal Serbia Seychelles Sierra Leone Singapore Slovakia Slovenia Solomon Islands Somalia South Africa South Georgia and The South Sandwich Islands Spain Sri Lanka Sudan Suriname Svalbard and Jan Mayen Swaziland Sweden Switzerland Syrian Arab Republic Taiwan Tajikistan Tanzania Thailand Timor-Leste Togo Tokelau Tonga Trinidad and Tobago Tunisia Turkey Turkmenistan Turks and Caicos Islands Tuvalu Uganda Ukraine United Arab Emirates US Minor Outlying Islands Uruguay Uzbekistan Vanuatu Venezuela Vietnam British Virgin Islands US Virgin Islands Wallis and Futuna Western Sahara Yemen Zambia Zimbabwe Kosovo Submit By clicking the Download Free Report button, you accept the terms and conditions and acknowledge that your data will be used as described in the GlobalData privacy policy By downloading this Report, you acknowledge that we may share your information with our white paper partners/sponsors who may contact you directly with information on their products and services. Visit our privacy policy for more information about our services, how we may use, process and share your personal data, including information on your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address. Thank you. Please check your email to download the Report.