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Company Directory

Use this alphabetized list to search for companies that provide information on single-patient expanded access. The web links in this directory are public information provided to the Foundation and the acknowledgement times listed are the number of days or hours for a company to acknowledge receipt of request, not to make a decision.

A |B |C |D |E |F |G |H |I |J |K |L |M |N |O |P |Q |R |S |T |U |V |W |X |Y |Z
Company Name
Phone Number & Email
Company Acknowledgement

Abbvie

Expected Application Timeframe
2 business days to acknowledge receipt

Alexion

Expected Application Timeframe
3 business days

Alkermes

Expected Application Timeframe
2 weeks

Additional Information

Alnylam Pharmaceuticals

Phone Number & Email
Expected Application Timeframe
3 business days

Additional Information

DISEASE/CATEGORY-SPECIFIC EA POLICIES/CRITERIA

http://www.alnylam.com/medical-professionals/expanded-access-program/

Amgen

Expected Application Timeframe
3 business days

AMICUS THERAPEUTICS

Expected Application Timeframe
3 business days

Additional Information

SINGLE-PATIENT EA POLICIES/CRITERIA
Requests for Early Access cannot be made directly by an individual patient or a patient’s parent/legal guardian or caregiver. Patients and caregivers seeking general information may reach out to Amicus’ Global Patient & Professional Advocacy Department at patientadvocacy@amicusrx.com or call toll-free in the United States at 1-866-9AMICUS (1-866-926-4287), or +44-1753-888-567 for International inquiries.

Astellas Pharma US

Expected Application Timeframe
7 days

AstraZeneca Pharmaceuticals LP

Expected Application Timeframe
Immediate

Biogen

Expected Application Timeframe
1 business day

Biohaven Pharmaceutical Holding Company Ltd.

Expected Application Timeframe
2 business days

Additional Information

SINGLE-PATIENT EA POLICIES/CRITERIA
Requests for Early Access must be made by a physician on behalf of their patient. A licensed physician overseeing the patient’s care, who is able to comply with Biohaven’s requirements, may contact Biohaven at BHV0223.ExpandedAccess@earlyaccesscare.com or call toll-free in the United States at 1-888-315-5797 (Option 6).

Boehringer Ingelheim Pharmaceuticals

Phone Number & Email
Expected Application Timeframe
5 business days

Bristol-Myers Squibb

Expected Application Timeframe
48 hours

Celgene

Phone Number & Email
Expected Application Timeframe
24 hours

Daiichi Sankyo

Expected Application Timeframe
10 business days

Eisai

Phone Number & Email
Expected Application Timeframe
2 business days

Eli Lilly

Phone Number & Email
Expected Application Timeframe
5 business days

EMD Serono

Expected Application Timeframe
5 business days

Genentech

Phone Number & Email
Expected Application Timeframe
3 business days

Additional Information

GlaxoSmithKline

Expected Application Timeframe
24 hours

Additional Information

SINGLE-PATIENT EA POLICIES/CRITERIA

http://www.gsk.com/media/3368/compassionate-use.pdf

Immix Biopharma

Phone Number & Email
Expected Application Timeframe
24hr to 48hr

Additional Information

Available therapies via single-patient EA

imx-110 - all advanced solid tumors

SINGLE-PATIENT EA POLICIES/CRITERIA

http://immixbio.com/pipeline/compassionate-use-policy/

Janssen

Expected Application Timeframe
Immediate

Additional Information

Johnson & Johnson

Kyowa Kirin Pharmaceutical Development, Inc.

Expected Application Timeframe
3 days

Additional Information

SINGLE-PATIENT EA POLICIES/CRITERIA
Poteligeo was approved by the FDA on August 8, 2018 for the treatment of adult patients with relapsed or refractory mycosis fungoides (MF) or Sézary syndrome (SS) after at least one prior systemic therapy. Only patients who have relapsed or refractory MF or SS after at least one prior systemic therapy will be eligible to participate.
The main purpose of the EA program is to facilitate the access of Poteligeo to patients who lack therapeutic alternatives and in which there is a reasonable expectation that the provision of access to Poteligeo will have a positive benefit/risk for the patient. The intention is for the EA program to fill the time between approval by the FDA and the date of availability of commercial supply to patients. The EA program will continue through November 1, 2018 when patients will transition to commercial supply.

Lundbeck

Phone Number & Email
Expected Application Timeframe
10 business days

Melinta Therapeutics, Inc.

Phone Number & Email
Expected Application Timeframe
Within 3 business days after receipt

Additional Information

Available therapies via single-patient EA

fusidic acid (CEM-102)

SINGLE-PATIENT EA POLICIES/CRITERIA
Consistent with Melinta Therapeutics’s mission to discover, develop, and commercialize a continual stream of novel antibiotics, we are focused on enrolling and conducting the clinical trials necessary to gain regulatory approvals and promptly make our medicines available to patients. At Melinta Therapeutics, we believe that wherever possible, the use of an investigational medicine for a patient as part of a clinical trial is preferable; however, when patients with an unmet medical need are unable to access our investigational products through a clinical trial and all other options have been exhausted, we may be able to provide access through our compassionate use or expanded access program.

The evaluation criteria to determine if Melinta Therapeutics can provide expanded access to a specific Melinta Therapeutics investigational agent outside of a clinical trial include all of the following:
• The patient has a serious or life-threatening illness or condition and is either no longer responsive to or no longer able to tolerate any available treatment option;
• The investigational drug is in active clinical development with sufficient data available to determine an appropriate dose and schedule for the patient’s specific condition;
• A benefit-risk analysis, based on both the available clinical data as well as the requesting physician’s assessment of the individual patient’s condition and history, supports making the investigational drug available;
• Access is compliant with local rules and laws;
• Making the investigational drug available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access; and
• Adequate supply of the investigational drug is available.

All requests must be submitted by the patient’s treating physician and Melinta Therapeutics may require more detailed information in order to fully evaluate a request. The treating physician must agree to obtain appropriate regulatory and ethics committee approvals and comply with regulatory obligations, including obtaining patient consent, patient monitoring and safety reporting. Treating physicians seeking expanded access, meeting the above-mentioned evaluation criteria, should submit their requests to medinfo@melinta.com. We regularly monitor this mailbox and will use our best efforts to acknowledge each submitted request within 3 business days after receipt. Melinta Therapeutics is committed to carefully evaluating requests on a case-by-case basis and in a fair and equitable manner; however, Melinta Therapeutics cannot guarantee access to any specific investigational drug by any individual patient.

If you are a patient with questions concerning access to our investigational products, either through a clinical trial or through expanded access, please consult with your treating physician.

Merck & Co.

Phone Number & Email

Millendo Therapeutics, Inc.

Novartis Pharmaceuticals

Expected Application Timeframe
Immediately

Additional Information

Available therapies via single-patient EA
Alpelisib - Alpha-specific PI3K inhibitor
Ceritinib - ALK+ treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) that is ALK-positive
Clofazimin - Pulmonary mycobacterium abscessus infection
Dabrafenib - Combo Trametinib in Melanoma
Eltrombopag - Chronic ITP, Congenital thrombocytopenia, Severe Aplastic Anemia, HCV patients requiring treatment with an interferon based therapy, or patients with low-and-int-1 MDS with severe thrombocytopenia
Everolimus - mTOR inhibition in Exploratory Development & special project
Lapatinib - HER2+ breast cancer
Nilotinib - CML
Panobinostat - Multiple Myeloma
Pasireotide - Exploratory/Tumor
Ribociclib - CDK4/6inhibitor

Novo Nordisk

Expected Application Timeframe
5 business days

Oncoceutics, Inc.

Expected Application Timeframe
Receipt will generally be acknowledged within 48 hours

Additional Information

Available therapies via single-patient EA

ONC201 - H3 K27M mutant gliomas

SINGLE-PATIENT EA POLICIES/CRITERIA
Oncoceutics will consider single - patient expanded access for patients who meet the criteria for the company's existing expanded access program, as outlined on clinicaltrials.gov

Otsuka America Pharmaceutical

Expected Application Timeframe
1 week

Pfizer

Expected Application Timeframe
5 business days

Additional Information

SINGLE-PATIENT EA POLICIES/CRITERIA

www.pfizercares.com

Retrophin

Expected Application Timeframe
7 business days

Sanofi

Expected Application Timeframe
1 business day

SCYNEXIS, Inc.

Expected Application Timeframe
SCYNEXIS will acknowledge a contact for requesting Expanded Access within 24 hours.

Additional Information

Available therapies via single-patient EA
SCY-078 is a triterpenoid, glucan synthase inhibitor, antifungal agent undergoing investigation for:
• The treatment of invasive candidiasis, including candidemia (IC).
• The treatment of invasive aspergillosis (IA), including IA that is refractory to or intolerant of other antifungal therapies.
• The treatment of invasive candidiasis in patients who are refractory to or intolerant of other antifungal therapies.
SINGLE-PATIENT EA POLICIES/CRITERIA
SCYNEXIS is committed to making Investigational Products available to seriously ill patients who have exhausted other treatment options. A treating physician, who is able to comply with the SCYNEXIS requirements , may request information about how to apply for access to SCYNEXIS’ Investigational Products by contacting the company.

Shire

Expected Application Timeframe
3 business days

Takeda Pharmaceuticals

Expected Application Timeframe
3 business days

Additional Information

DISEASE/CATEGORY-SPECIFIC EA POLICIES/CRITERIA

http://takedaoncology.com/medicines/access-to-investigational-medicines

Teva

Expected Application Timeframe
1 business day

Additional Information

SINGLE-PATIENT EA POLICIES/CRITERIA

http://www.tevapharm.com/files/policy.pdf

UCB

Phone Number & Email

Additional Information

Ultragenyx

Additional Information

SINGLE-PATIENT EA POLICIES/CRITERIA
Requests for early access to investigational therapies must be made by a qualified physician.