European Commission Grants Orphan Drug Designation for Nirogacestat for the Treatment of Soft Tissue Sarcoma.

Nirogacestat is a selective oral small molecule, gamma-secretase inhibitor under development by SpringWork Therapeutics. Currently, in Phase III development, the drug is investigated for the treatment of progressive Desmoid tumors, which are a type of soft-tissue tumour.

The drug has already been recommended by the FDA in June 2018.

Desmoid tumors are an abnormal increase in the connective tissues. The connective tissues are responsible for the strength and flexibility of the bones, ligaments, and muscles. These tumors are considered locally invasive but non-metastasizing.

Desmoid Tumors account for less than 3% of soft tissue tumors. According to, the annual Desmoid tumors incidence is estimated to range between 1/250,000-1/500,000. These tumors more predominantly affect women between the ages of 15-60 years.

Soft Tissue Sarcomas (STS) is a family of rare, mesenchymal (connective tissue) neoplasms with heterogeneous differentiation and may arise in various organs (including fat, muscle, nerve and nerve sheath, blood vessels, and other connective tissue).

As per the American Cancer Society’s estimates, a slight male preponderance is observed over the female, although this difference is rarely statistically significant.

These tumors develop mainly in adults around the age of 50, the frequency increases with age; and 10% of patients are children and adolescents.

The market outlook and treatment landscape of STS have changed dramatically in the last decades, with advances in the diagnosis of subtyping and with the introduction of targeted therapies. The treatment market is segmented into surgery, radiation therapy, chemotherapy, anti-angiogenesis drugs and targeted therapy.

Sarcoma treatment landscape has changed dramatically in the last few years with the introduction of targeted therapies.

Evotec- Germany based pharma company has entered into a collaboration with Japan’s Takeda Pharmaceutical for the development of at least five drug discovery programs across a broad range of indications.

Under the terms of the agreement, Evotec will use its integrated drug discovery program to advance Takeda’s therapeutic areas namely oncology, gastroenterology, neuroscience and rare diseases.

Evotec is planning to use the drug discovery platform to accelerate small molecule programs. Once Evotec is able to produce a drug candidate, Takeda will have the option to fully acquire it.

Furthermore, Takeda will pay Evotec the amount upfront one-time to gain access to the latter’s platform. Whereas, Evotec is eligible to gain an amount of USD 170 million for achieving each milestone in addition to realities on the sales.

The US FDA has given its recommendation for Jynneos Smallpox and Monkeypox Vaccine, to be used for the prevention of monkeypox and smallpox in adults aged 18 years and above.

Manufactured by Bavarian Nordic, this is the only present vaccine which is FDA-approved for the prevention of monkeypox and smallpox.

Smallpox is a viral infection caused by Variola virus with fatality rates approximately 30% overall, although survivors frequently suffered significant morbidity including blindness and skin scarring, as per NCBI.

Smallpox is in existence for over 3,000 years and was considered the global threat until it was eradicated by a collective global vaccination programme led by the World Health Organization.

However, it still remains the most feared disease due to the nature of a highly contagious virus.

The monkeypox virus is similar to human smallpox, however, monkeypox virus is much trivial than smallpox, but it can be fatal.

The vaccine will be administered to people who are at high risk of either of the diseases.

Jynneos vaccine is a part of the Strategic National Stockpile (SNS), which will ensure it easy and convenient accessibility to the masses in the US.