List of the Compassionate Use programs of the Paul-Ehrlich Institut

Stand 13.09.2021

Medicinal Product: Anifrolumab Active substance Anifrolumab Pharmaceutical Form Concentrate for solution for infusion Application Intravenous infusion Dosing 300 mg Anifrolumab, administered as an intravenous infusion over a 30 minute period, every 4 weeks Indication As an add-on therapy for the treatment of adult patients with moderate to severe systemic erythematosus (SLE), despite standard therapy Date of Confirmed 15.03.2021 Notification Duration of 14.03.2022 Compassionate Use Programme Responsible Person AstraZeneca GmbH, (Company), Contact Tinsdaler Weg 183, Person 22880 Wedel, Germany Tel.:0800/22 88 660; E-Mail: [email protected]

Medicinal Product: Active substance Monoclonal antibody Lanadelumab Pharmaceutical Form Concentrate for solution for infusion Application Subcutaneous injection Dosing 150 mg Lanadelumab, administered as a subcutaneous injection. 150 mg dose given either every 2 weeks or 4 weeks Indication Prevention of acute attacks of hereditary angioedema (HAE) in pediatric patients 2 to <12 years of age. To be eligible patients must have completed Study SHP643- 301. Date of Confirmed 30.03.2021 Notification Duration of 29.03.2022 Compassionate Use Programme Responsible Person Responsible person: (Company), Contact Takeda Development Center Americas, Inc (TDC Americas) Person 95 Hayden Avenue, Lexington, MA 02421, USA

Contact person: Marcelo Freire

Paul-Ehrlich-Institut Paul-Ehrlich-Str. 51-59 63225 Langen, Germany >>www.pei.de Takeda Italia S.p.A Via Elio Vittorini 129 00144 ROMA ITALIA Email: [email protected] Telephone: +41 79 758 4815

Medicinal Product: Amivantamab Active substance Monoclonal antibody Amivantamab Pharmaceutical Form Concentrate for solution for infusion 7ml vial contains 350 mg of Amivantamab (50 mg Amivantamab per ml) Application Intravenous use Dosing The recommended dose of Amivantamab is 1.050 mg for patients with a bodyweightless than 80 kg and 1.400 mg for patients with a bodyweight greater than or equal to 80 kg. Amivantamab should be administered intravenously once weekly for the first 4 weeks (1 cycle=28 days), then every 2 weeks thereafter. The first dose is split over two days.

Indication As monotherapy for the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with an EGFR Exon 20 insertion mutation after failure of platinum-based chemotherapy Date of Confirmed 15.04.2021 Notification Duration of 14.04.2022 Compassionate Use Programme Responsible Person Janssen Pharmaceutica NV; Turnhoutseweg 30; (Company), Contact B-2340 Beerse,Belgium Person Contact in Germany: Janssen-Cilag GmbH, Medical Service Center E-Mail: [email protected] Tel.: +49 2137-955-955 Fax.: +49 2137-955-443

Medicinal Product: Retifanlimab Active substance Monoclonal antibody Retifanlimab Pharmaceutical Form Concentrate for infusion solution, 25mg/ml Application Intravenous administration Dosing Intravenous administration over 30 minutes (-5/+15 min) 500 mg Q4W

Seite 2/6 Indication Retifanlimab is indicated for the treatment of patients with squamous carcinoma of the anal canal (SCAC) who have progressed following platinum- based chemotherapy Date of Confirmed 11.05.2021 Notification Duration of 10.05.2022 Compassionate Use Programme Responsible Person Incyte Biosciences International SARL (Company), Contact Rue Docteur-Yersin 10, CH-1110 Morges, Person Switzerland Dr. Karl Boegl, MD PhD MBA Email: [email protected]

EU Local Representative: Dr. Katrin Edmaier-Schröger, PhD Position: Country Medical Director Function/Relationship to RP: Employee Address: Incyte Biosciences Germany GmbH, Fraunhoferstraße 9 D-82152 Planegg/Martinsried Email: [email protected] Phone: +49 89 203010 855

Medicinal Product: Sacituzumab Govitecan (IMMU-132) Active substance Monoclonal antibody Sacituzumab Govitecan Pharmaceutical Form Powder for Concentrate for Solution for Infusion Application Intravenous use Dosing The recommended dose is 10 mg/kg administered as an intravenous infusion once weekly on Days 1 and 8 of 21-day treatment cycles. Indication Unresectable locally-advanced or metastatic Triple-Negative Breast Cancer for which patient have received at least two prior therapies, including at least one prior therapy for locally- advanced or metastatic disease Date of Confirmed 21.05.2021 Notification Duration of 20.05.2022 Compassionate Use Programme Responsible Person Responsible Person: (Company), Contact Gilead Sciences, Inc. Person 333 Lakeside Drive Foster City, CA 94404 USA

EU Representative: Gilead Sciences Ireland UC

Seite 3/6 IDA Business & Technology Park Carrigtohill Co. Cork Ireland

Contact Details: [email protected]

Medicinal Product: Tebentafusp (IMCgp100) Active substance Fusionprotein Tebentafusp (IMCgp100) Pharmaceutical Form Concentrate for solution for infusion Application Intravenous infusion Dosing 20 mcg C1D1; 30 mcg C1D8; 68 mcg C1D15 and subsequent doses (Every week: Days 1, 8, and 15 of 21-day cycle) Indication HLA-A*02:01 positive patients with metastatic uveal melanoma (mUM) Date of Confirmed 08.06.2021 Notification Duration of 07.06.2022 Compassionate Use Programme Responsible Person Immunocore, Ltd. (Company), Contact 101 Park Drive, Milton Park Abingdon, OX14 4RY United Person Kingdom

EU Local Representative: Dr Jessica Hassel, Universitaetsklinikum Heidelberg Im Neuenheimer Feld 672 69120 Heidelberg Germany Phone: +4962215638503 Fax: +496221564798 [email protected]

Medicinal Product: Efgartigimod Active substance Monoclonal antibody Efgartigimod Pharmaceutical Form concentrate for solution for infusion 20 mg/mL Application Intravenous administration Dosing Dosage Level(s): Efgartigimod IV 10 mg/kg

Administer efgartigimod in treatment cycles of once weekly infusions for 4 weeks.

Initiate subsequent treatment cycles ≥ 7 weeks from the initial infusion of the previous cycle based on clinical evaluation Indication Adult Patients With Generalized Myasthenia Gravis

Seite 4/6 All of the following criteria must apply for the patient to be eligible for the program:

1. The patient must have MGFA disease class IIIb, IVa, or IVb at the time of inclusion 2. The patient is refractory to existing treatments, meeting one of the following criteria: a. Insufficient response (eg, persistent moderate to severe weakness) to maximal safe doses of steroids and at least one immunosuppressive drug at adequate dose and duration b. Severe or intolerable adverse effects from immunosuppressive therapy in inclusion criterion 2a c. Comorbid conditions restricting the use of therapies in inclusion criterion 2a d. History of frequent life-threatening events caused by myasthenia gravis (eg, myasthenic crisis; pneumonia due to dysphagia and immunosuppressive treatment) 3. In addition, insufficient clinical response (eg, persistent moderate to severe weakness), or intolerable side effects, patient not clinically eligible or contraindications (based on treating physician’s decision) to treatment with therapies such as (for AChR-Ab positive patients), regular IVIg, PLEX or immunoadsorption, or rituximab 4. The patient is not eligible for or able to participate in a clinical trial (based on the treating physician’s decision)

Date of Confirmed 21.06.2021 Notification Duration of 20.06.2022 Compassionate Use Programme Responsible Person Details of the Responsible Person (RP): (Company), Contact Name : argenx BV Person Address: Industriepark 7, B-9052 Zwijnaarde, Belgium

Details of the Contact Person (CP): Name: Jana Podhorna MD, Ph.D. Position: Senior Medical Director Function/Relationship to RP: Employee Adresse: Industriepark 7, 9052 Zwijnaarde, Belgien Telephone: +43 676 9335422 Email: [email protected]

Medicinal Product: Spesolimab Active substance Monoclonal antibody Spesolimab Pharmaceutical Form Solution for infusion Application Intravenous infusion Dosing 900 mg intravenous (i.v.), i.e. 2 vials. Single dose treatment.

Seite 5/6 If deemed necessary by the treating physician (i.e., if the flare symptoms persist), a second dose of 900 mg spesolimab may be administered one week after the initial infusion Indication Treatment of generalized pustular psoriasis (GPP) presenting with a flare for patients for whom no satisfactory authorized alternative therapy exists (“high medical unmet need“)

Date of Confirmed 03.08.2021 Notification Duration of 02.08.2022 Compassionate Use Programme Responsible Person Elmar Daalman (Company), Contact Boehringer Ingelheimer Pharma GmbH & Co. KG Person Binger Straße 173 55216 Ingelheim am Rhein E-Mail: [email protected] Tel.: +49 6132 77 90338 Fax: +49 6132 72 93310

Medicinal Product: Padcev (Enfortumab Vedotin) Active substance Monoclonal antibody Enfortumab Vedotin Pharmaceutical Form Powder for concentrate for solution for infusion Application Intravenous infusion Dosing The recommended dose of enfortumab vedotin is 1.25 mg/kg (up to a maximum of 125 mg for patients ≥ 100 kg) administered as an intravenous infusion over 30 minutes on Days 1, 8 and 15 of a 28-day cycle until disease progression or unacceptable toxicity Indication Treatment of adult patients with locally advanced or metastatic urothelial cancer who have received a platinum-containing chemotherapy and a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor Date of Confirmed 06.08.2021 Notification Duration of 05.08.2022 Compassionate Use Programme Responsible Person Responsible Person: (Company), Contact Astellas Pharma Europe B.V., Sylviusweg 62, 2333 BE Person Leiden, The Netherlands

Contact Details: [email protected]

Kontakt: [email protected]

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