orphan drug status

FDA grants orphan drug designation to nadunolimab for pancreatic cancer

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The FDA granted orphan drug designation to nadunolimab for the treatment of pancreatic cancer.

Nadunolimab (CAN04, Cantargia AB) is an interleukin-1 receptor accessory protein (IL1RAP)-binding antibody in development for various types of cancer.

FDA sign
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Two trials are assessing the agent for pancreatic ductal adenocarcinoma, which accounts for more than 90% of pancreatic cancer cases.

The phase 1/phase 2A CANFOUR study is evaluating nadunolimab in combination with gemcitabine and nab-paclitaxel (Abraxane, Bristol Myers Squibb) as first-line treatment. Interim efficacy data from 33 patients showed the regimen led to longer PFS and OS than historical control data.

The phase 1B CAPAFOUR trial is evaluating nadunolimab with FOLFIRINOX as first-line therapy for patients with metastatic pancreatic ductal adenocarcinoma.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.

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To determine whether a general societal preference for prioritising treatment of rare diseases over common ones exists and could provide a justification for accepting higher cost effectiveness thresholds for orphan drugs.

Design Cross sectional survey using a web based questionnaire.

Setting Norway.

Participants Random sample of 1547 Norwegians aged 40-67.

Main outcome measure Choice between funding treatment for a rare disease versus a common disease and how funds should be allocated if it were not possible to treat all patients, for each of two scenarios: identical treatment costs per patient and higher costs for the rare disease. Respondents rated five statements concerning attitudes to equity on a five point Likert scale (5=completely agree).

Results For the equal cost scenario, 11.2% (9.6% to 12.8%) of respondents favoured treating the rare disease, 24.9% (21.7% to 26.0%) the common disease, and 64.9% (62.6% to 67.3%) were indifferent. When the rare disease was four times more costly to treat, the results were, respectively, 7.4% (6.1% to 8.7%), 45.3% (42.8% to 47.8%), and 47.3% (44.8% to 49.8%). Rankings for attitude on a Likert scale indicated strong support for the statements “rare disease patients should have the right to treatment even if more expensive” (mean score 4.5, SD 0.86) and “resources should be used to provide the greatest possible health benefits” (3.9, 1.23).

Conclusions Despite strong general support for statements expressing a desire for equal treatment rights for patients with rare diseases, there was little evidence that a societal preference for rarity exists if treatment of patients with rare diseases is at the expense of treatment of those with common diseases.

source:BM J