Bayer

Tumors Harboring NTRK Fusion

Larotrectinib expanded access is for patients with cancer with a NTRK1, NTRK2, or NTRK3 gene fusion, who are ineligible for an ongoing larotrectinib clinical trial or have other considerations that prevent access to larotrectinib through an existing clinical trial. Gene fusion occurs when a gene is made by joining parts of two different genes. NTRK gene fusion can lead to the development of solid tumors in a variety of tissue types. The study drug larotrectinib blocks the action of the NTRK gene fusion. Expanded access is intended to treat individual patients with different types of cancers with a NTRK gene fusion who are unresponsive to current standard treatment for their condition and also are unable to participate in ongoing clinical trials.

Solid Tumors Harboring NTRK Fusion

Selitrectinib expanded access is for minor and adult patients with cancer having a change in a particular gene (NTRK1, NTRK2, or NTRK3 gene fusion). The patients are ineligible for an ongoing selitrectinib clinical trial or have other considerations that prevent access to selitrectinib through an existing clinical trial. Expanded access is intended to treat individual patients with different types of cancers with a NTRK gene fusion, including blood cancers, who have previously received tropomyosin receptor kinase (TRK) inhibitor therapy.

AstraZeneca Pharmaceuticals LP

Advanced Non-Small Cell Lung Cancer
Metastatic Non Small Cell Lung Cancer

The purpose of this Medical Access Program (also referred to as an Expanded Access Program in the USA) is to provide access to Dato-DXd for eligible patients with previously treated advanced or metastatic nonsquamous non-small cell lung cancer (NSCLC) who, in their treating physician's opinion, have an unmet clinical need, are unlikely to obtain optimal benefit from currently approved and commercially available drugs, and who cannot enter a suitable clinical trial.

Unresectable Hepatocellular Carcinoma

To provide early access (i.e., before marketing authorisation) to tremelimumab 300 mg IV administered once on Day 1 of Cycle 1 plus durvalumab 1500 mg IV followed by durvalumab 1500 mg IV Q4W monotherapy in patients with unresectable HCC.

Non Small Cell Lung Cancer
Cancer of the Head and Neck

The purpose of this study is to provide ZD1839 for those patients with locally advanced and/or metastatic non-operable non-small cell lung cancer (stage III or IV) or recurrent and/or metastatic squamous cell head and neck cancer who receive the therapy on an expanded access basis due to their inability to meet eligibility criteria for on-going recruiting trials, inability to participate in other clinical trials (e.g., poor performance status, lack of geographic proximity), or because other medical interventions are not considered appropriate or acceptable.

Non Small Cell Lung Cancer (NSCLC)

The purpose of this study is to provide gefitinib treatment to patients who, on completion or closure of other gefitinib clinical studies, were either receiving placebo treatment, or are continuing on the same dose and regimen of gefitinib established in their preceding study, for as long as the patients continue to derive benefit.

Ovarian Cancer

This is an open-label, single-arm, international, multicenter Multiple Patient Expanded Access Program (MPEAP). The program is designed to provide treatment access to olaparib tablets for patients with platinum-sensitive relapsed high-grade epithelial ovarian, fallopian tube or primary peritoneal cancer without other treatment options or eligible for an olaparib clinical trials.

NF type1 With Inoperable Plexiform Neurofibromas

This will be an open-label, single-arm, multicenter intermediate access protocol which provides treatment access to selumetinib for eligible patients with neurofibromatosis type 1 (NF1) who have inoperable, progressive/symptomatic plexiform neurofibromas (PN) without any alternative therapeutic options. All patients will continue to receive drug while they are deriving clinical benefit. Approximately 100 patients in the US will be treated as part of this protocol

Relapsed/Refractory Hairy Cell Leukemia

Early Access Programme to provide treatment access to moxetumomab pasudotox for eligible patients with relapsed/refractory hairy cell leukemia

Astellas Pharma US

Locally Advanced Unresectable Gastroesophageal Junction (GEJ) Adenocarcinoma Cancer
Locally Advanced Unresectable Gastric Adenocarcinoma Cancer
Metastatic Gastric Adenocarcinoma Cancer
Metastatic Gastroesophageal Junction (GEJ) Adenocarcinoma

This program is to provide zolbetuximab to people with stomach cancer or gastroesophageal junction (the junction between stomach and esophagus) cancer who have not yet been treated with chemotherapy, immunotherapy, or zolbetuximab and who have tested positive for claudin18.2 (a protein found in some cancer cells). People will work with their doctor to see if they are capable of being treated with zolbetuximab while they receive other standard medicines to treat their cancer. The program will allow people early access to zolbetuximab before the drug is fully approved. Zolbetuximab will be given through a vein. This is called an infusion. The infusion will happen during a person's treatment with other cancer medicines. Zolbetuximab will be given every 3 weeks. People will continue treatment until: they have medical problems (unwanted side effects) from the treatment; their cancer gets worse; they start other cancer treatment; they ask to stop treatment; or they do not come back for treatment. People will visit the clinic on certain days during their treatment. During these visits, the program doctors will check for any medical problems (unwanted side effects) from zolbetuximab, other cancer treatment, or both. At some visits, other checks will include a medical examination, laboratory tests and vital signs. Vital signs include temperature, pulse, and blood pressure. Also, blood and urine samples will be taken. People will visit the clinic within 7 days after stopping treatment. The program doctors will check for any medical problems (unwanted side effects) from zolbetuximab or their cancer treatment. Other checks will include a medical examination, laboratory tests, and vital signs. Then, people will have a follow-up visit about 30 days after stopping treatment. If people are having no health problems, the follow-up visit can happen over the telephone.

Metastatic Castration-Resistant Prostate Cancer

The purpose of this treatment protocol is to provide expanded access to MDV3100 and monitor its safety in patients with metastatic castration-resistant prostate cancer previously treated with docetaxel-based chemotherapy.

Acute Myeloid Leukemia (AML)
FMS-like Tyrosine Kinase-3 (FLT3) Mutations

The purpose of this study is to provide expanded access to ASP2215 for subjects with FLT3-mutated relapsed or refractory AML or FLT3-mutated AML in composite complete remission (CRc) (complete remission [CR], complete remission with incomplete hematologic recovery [CRi], complete remission with incomplete platelet recovery [CRp]) with MRD without access to comparable or alternative therapy.

Amicus Therapeutics

Fabry Disease

This program allows physicians to request permission from Amicus Therapeutics (Amicus) for treatment access to migalastat hydrochloride (HCl) for specific adult patients with Fabry disease. Treatment is open label for 6 months with renewal every 6 months.

Pompe Disease

This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.

Pompe Disease Infantile-Onset

This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.